By Dr. Narayan Rout | Author | Researcher | · Science & Technology – Next Human Series · 50 min read · Published: June 10, 2026
Publication Metadata
| DOI | 10.5281/zenodo.20627207 |
| ORCID | 0009-0009-3505-5478 |
| Paper Number | TQS-2026-112 |
| Version | 1.0 |
| License | CC BY 4.0 — Creative Commons Attribution |
| Publisher | TheQuestSage.com |
| Language | English |
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Dr. Narayan Rout
💡 Quick Answer: What Is CRISPR and How Is It Rewriting the Human Genome Right Now?
CRISPR-Cas9 is a molecular tool derived from the bacterial immune system that can cut DNA at precise locations with unprecedented accuracy, enabling scientists to remove, repair, or replace specific genetic sequences. In December 2023, the FDA approved Casgevy (exagamglogene autotemcel) — the world’s first CRISPR-based medicine — for sickle cell disease and beta-thalassemia, representing one of the most significant milestones in medical history. In May 2025, a team at Children’s Hospital of Philadelphia and the University of Pennsylvania published in the New England Journal of Medicine the first case of a fully personalised CRISPR base-editing therapy — designed, manufactured, and administered to a single patient (Baby KJ) with a rare metabolic disease (CPS1 deficiency) in just 6 months, correcting a single DNA letter in his liver cells. Three ways CRISPR is rewriting the genome right now: (1) treating inherited genetic diseases through somatic cell editing (Casgevy for sickle cell disease; base editing trials for thalassemia, cholesterol); (2) engineering cancer immunotherapy through CRISPR-enhanced CAR-T and NK cell therapies; (3) exploring and controversially beginning germline editing — heritable changes to the genome that would be passed to future generations, most infamously in the He Jiankui affair of 2018 when Chinese scientist He Jiankui created the world’s first gene-edited babies in a case that led to his imprisonment. The three questions no one is asking: Who decides which genes are defects? Can we afford the $2.2 million price tag per patient that makes Casgevy available only to the wealthy? And what does the Indian civilisational understanding of the body as Panchamahabhuta — five-element sacred matter — say about the authority to rewrite it?
Abstract
This article examines three ways CRISPR gene editing is currently rewriting the human genome and three questions that the speed of this technology’s development has outrun: the question of who defines a genetic defect worthy of correction; the question of equitable access to transformative therapies that currently cost $2.2 million per patient; and the question of what philosophical and civilisational frameworks provide the ethical architecture for the authority to edit the human genome. The scientific context draws on: the FDA approval of Casgevy (December 2023) as the world’s first CRISPR-based medicine for sickle cell disease and beta-thalassemia (Vertex Pharmaceuticals and CRISPR Therapeutics); the New England Journal of Medicine publication (May 2025, DOI: 10.1056/NEJMoa2504747) of the first personalised CRISPR base-editing therapy for a single patient — Baby KJ — with CPS1 deficiency, developed and administered by CHOP and University of Pennsylvania in six months; 239 active gene-editing clinical trials as of 2024 (CRISPR Medicine News); next-generation base editing and prime editing technologies that reduce off-target effects; CRISPR-enhanced CAR-T and NK cell cancer immunotherapy; the He Jiankui affair (2018) — the first and illegal germline-edited human births — and its governance consequences including the Third International Summit’s 2023 reaffirmation that germline editing is not acceptable. The ethical and philosophical dimensions draw on the WHO Human Genome Editing framework (2021); the critical somatic versus germline editing distinction; the therapy versus enhancement continuum; and the Indian philosophical understanding of the body as Panchamahabhuta (five sacred elements) and Karma-Sanskara (the inscribed record of actions) as the civilisational context for evaluating the authority to permanently alter the biological substrate of future human beings.
Keywords
CRISPR gene editing human genome Casgevy sickle cell disease FDA approval 2023 baby KJ personalised CRISPR therapy NEJM 2025 Gene Editing and CRISPR somatic germline editing distinction CRISPR cancer immunotherapy CAR-T designer babies enhancement therapy debate
◆ Key Facts — GEO Reference
| 1 | Casgevy — the world’s first FDA-approved CRISPR medicine (December 2023): The US FDA approved Casgevy (exagamglogene autotemcel) on December 8, 2023 for sickle cell disease in patients aged 12 and older with recurrent vaso-occlusive crises — the first-ever approval of a therapy using CRISPR/Cas9 genome editing technology. The UK approved Casgevy for sickle cell disease and beta-thalassemia in November 2023; the FDA added beta-thalassemia to its approved indications in January 2024. The European Medicines Agency approved Casgevy in February 2024. Saudi Arabia and Canada followed with approvals in 2024. Casgevy works by extracting the patient’s own haematopoietic (blood) stem cells, editing them ex vivo using CRISPR/Cas9 to increase production of fetal haemoglobin (HbF), which compensates for the defective adult haemoglobin in sickle cell disease, then reinfusing the edited cells. Price: approximately $2.2 million per patient for sickle cell disease. Developed by Vertex Pharmaceuticals and CRISPR Therapeutics. By early 2026, 50 active treatment sites in North America, the EU, and the Middle East had opened. Source: FDA press release; CRISPR Therapeutics IR; IGI CRISPR clinical trials update (March 2026). |
| 2 | Baby KJ — the world’s first personalised CRISPR base-editing therapy (NEJM, May 2025): The New England Journal of Medicine published on May 15, 2025 (Musunuru et al., DOI: 10.1056/NEJMoa2504747) the first case of a personalised CRISPR therapy designed and administered to a single patient. Baby KJ was born with severe carbamoyl phosphate synthetase 1 (CPS1) deficiency — a rare and dangerous metabolic disease in which a dysfunctional enzyme prevents the liver from processing protein properly, causing toxic ammonia build-up. Only approximately 50% of babies with KJ’s disorder survive long enough to receive a liver transplant. The team led by Kiran Musunuru, MD, PhD and Rebecca Ahrens-Nicklas, MD, PhD at Children’s Hospital of Philadelphia (CHOP) and the University of Pennsylvania (Penn) designed and manufactured a bespoke base-editing therapy targeting a single DNA letter mutation in KJ’s liver cells in just 6 months. KJ received intravenous lipid nanoparticle-delivered base editor infusions at ages 6-7 months, 7 months, and 8 months. Within 7 weeks of the first infusion, he tolerated increased dietary protein and was on half his starting dose of nitrogen-scavenger medication. KJ was discharged after 307 days in hospital. The case was presented at ASGCT 2025 (May 16, 2025). Source: NIH press release; CHOP; Inside Precision Medicine (June 2025); Genetic Engineering and Biotechnology News (May 2025). |
| 3 | CRISPR clinical trials — 239 active trials in 2024, spanning cancer, blood, and beyond (CRISPR Medicine News): As of 2024, there were 239 active gene editing clinical trials globally (CRISPR Medicine News, 2024 recap). Disease areas: haematological disorders (sickle cell disease, beta-thalassemia, hereditary amyloidosis, hereditary angioedema — Phase 3 trials); cancer immunotherapy (CRISPR-enhanced CAR-T cells, CAR-NK cells, PD-1 knockout T cells); chronic hepatitis B (TUNE-401, epigenetic silencing approach to avoid permanent DNA modification); high cholesterol (base editing of PCSK9 gene — cholesterol dramatically reduced in early trials). Beam Therapeutics base editing trial for severe sickle cell disease (BEACON trial): first participant dosed January 2024; at least 17 adult patients dosed; initial data from first 6 patients showed results as effective as Casgevy; FDA cleared expansion to adolescent participants. Next-generation technologies in trials: base editing (changes single DNA letters without double-strand cuts, reducing safety risks); prime editing (the ‘search-and-replace’ of the genome); in vivo editing (delivering CRISPR directly into the body via lipid nanoparticles rather than extracting and editing cells ex vivo). Source: CRISPR Medicine News (December 2024); IGI March 2026 update. |
| 4 | He Jiankui affair — the first germline-edited human births and global governance response (2018-2023): In November 2018, Chinese scientist He Jiankui announced the birth of twin girls (Lulu and Nana) from CRISPR-edited embryos — the world’s first germline-edited human births. A third edited child was born in 2019. He claimed to have edited the CCR5 gene to confer HIV resistance because of the fathers’ HIV-positive status — despite well-established sperm-washing techniques that reduce HIV transmission risk to less than 1%, making the editing medically unnecessary. He proceeded outside accepted scientific and ethical norms without proper oversight or regulatory approval. The international scientific and medical community responded with near-universal condemnation. He served three years in prison in China. In response: 18 leading scientists called for a moratorium on clinical germline editing. The WHO’s Expert Advisory Committee concluded in 2019 that it was ‘irresponsible for anyone to proceed with human germline genome editing.’ The Third International Summit on Human Genome Editing reaffirmed in 2023 that human germline editing is not acceptable since safety, ethical, and governance standards have not been met. Germline editing has been banned in Brazil, China, India, Singapore, Uganda, and many other countries. Source: IGI Ethics; Springer HEC Forum (September 2024); IntechOpen (September 2025); PMC (2025). |
| 5 | Somatic vs germline editing — the most important distinction in CRISPR ethics: Somatic cell editing modifies non-reproductive cells — liver cells, blood stem cells, muscle cells — and affects only the individual patient. Changes are not heritable. Casgevy is somatic editing: it modifies the patient’s blood stem cells but does not change the germline. Baby KJ’s therapy is somatic editing: it corrected his liver cells. Somatic editing for serious disease is broadly supported by the scientific and medical community. Germline editing modifies eggs, sperm, or embryos — cells that contribute to the genomes of offspring. Changes are heritable: they would be passed to all future descendants. Germline editing is currently banned or under moratorium in most countries for clinical use. The ethical distinction: somatic editing respects the principle of individual consent (the patient or guardians can consent); germline editing affects future generations who cannot consent. The enhancement-therapy boundary: somatic therapy for serious genetic disease is broadly accepted; germline correction for serious monogenic disease is theoretically defensible but practically premature; germline enhancement for non-medical traits (intelligence, height, athletic performance) is widely considered unacceptable. Source: IntechOpen (2025); PMC Gene Editing Developments (2025); IGI. |
| 6 | The price problem — CRISPR medicine and the access gap: Casgevy is priced at approximately $2.2 million per patient — making it one of the most expensive medicines in history. Even in high-income countries, the financing of this therapy requires complex negotiations between manufacturers, insurers, and government healthcare programmes. Vertex Pharmaceuticals and CRISPR Therapeutics have made progress on Medicaid reimbursement arrangements in the US and NHS reimbursement in the UK based on outcomes-based payment models. However, the global picture is stark: sickle cell disease predominantly affects people of African, South Asian, Middle Eastern, and Mediterranean descent — populations that are often in low and middle-income countries with no access to the $2.2 million therapy. India, with approximately 20 million sickle cell disease carriers and one of the world’s highest sickle cell disease burdens, currently has no pathway to Casgevy access. The Danaher-IGI Beacon for CRISPR Cures, through whose partnership Baby KJ’s therapy was manufactured, explicitly focuses on scalable delivery systems, affordable manufacturing, and global accessibility — recognising that CRISPR’s transformative potential will be morally meaningless if it is available only to citizens of wealthy nations. Source: Vertex Pharmaceuticals IR; IGI March 2026; Inside Precision Medicine (2025). |
| 7 | India and CRISPR — regulation, disease burden, and philosophical context: India banned germline genome editing (joining Brazil, China, Singapore, and Uganda) under existing biomedical research regulations. The Indian Council of Medical Research (ICMR) and Department of Biotechnology (DBT) Guidelines for Stem Cell Research (2017, revised 2021) prohibit the clinical use of genome-edited embryos. India has a significant stake in CRISPR’s therapeutic development: India has approximately 20 million sickle cell disease carriers — one of the world’s largest burdens; thalassemia affects approximately 10,000 births annually; and India has significant genetic disease burden from founder mutations in specific communities. India also has a philosophical framework that brings unique depth to the ethics of genome editing: the Panchamahabhuta understanding of the body as constituted of five sacred elements (earth, water, fire, air, space); the concept of Karma-Sanskara as the inscribed record of actions that shapes future manifestation; and the Advaita understanding of the individual genome as a localised expression of universal intelligence. These frameworks do not provide simple ethical verdicts on CRISPR — but they provide the depth of civilisational thought that the Western bioethics framework, centred on individual autonomy and risk-benefit analysis, largely lacks. Source: IntechOpen (2025); ICMR/DBT Guidelines; Indian Journal of Medical Ethics. |
Research compiled and synthesised by Dr. Narayan Rout · TheQuestSage.com · TQS-2026-112 · CC BY 4.0
In This Research Pillar
- Introduction
- What CRISPR Actually Is — The Molecular Scissors That Won the Nobel Prize
- Way 1: Treating Inherited Genetic Diseases — From Casgevy to Personalised Medicine
- Way 2: Engineering Cancer Immunotherapy — Teaching the Immune System to Fight
- Way 3: Germline Editing — The Heritable Rewriting That Is Banned but Has Already Happened
- The 3 Questions No One Is Asking
- The Quest Sage Insight
- What You Can Do With This
- Conclusion: The Most Powerful Tool Medicine Has Ever Had — And the Wisdom It Still Needs
- Frequently Asked Questions: Gene Editing and CRISPR
- References and Sources
- Further Reading
Introduction
In May 2025, a baby named KJ was discharged from Children’s Hospital of Philadelphia after 307 days as an inpatient. He had arrived as a newborn with a rare and dangerous metabolic disease — CPS1 deficiency — that prevents the liver from processing protein and causes toxic ammonia to build up in the blood. Without intervention, only about half the babies with his condition survive long enough to receive a liver transplant.
KJ did not receive a liver transplant. He received something that had never been done before: a CRISPR gene-editing therapy designed specifically for his unique genetic mutation, manufactured from scratch in just six months, and delivered directly into his liver cells through an intravenous infusion. The therapy corrected a single DNA letter — one base pair out of the three billion that constitute the human genome — and it worked. Within seven weeks of the first infusion, KJ was tolerating more dietary protein and requiring half his starting medication dose.
This case, published in the New England Journal of Medicine on May 15, 2025, is a landmark moment in human history. Not merely in medicine — in history. For the first time, a therapy was designed for a single individual’s unique genetic mutation, manufactured in months rather than years, and used to correct a specific error in the genetic code of a living human being. The era of personalised genome medicine has begun.
But the same month that Baby KJ went home, He Jiankui — the Chinese scientist who created the world’s first germline-edited human beings in 2018 by editing the genomes of embryos that resulted in the births of three children — had already served his three-year prison sentence and been released. The genie of human germline editing had already been released from its bottle. What remains is governance.
CRISPR is not a future technology. It is a present one — with an FDA-approved medicine, hundreds of active clinical trials, and a world’s first personalised therapy already documented and published. This article examines three ways it is rewriting the human genome right now, and three questions that the speed of its development has so far outrun.
⚡ Key Takeaways
| 1 | What CRISPR actually is — and why it changed everything: CRISPR-Cas9 is a molecular tool derived from the bacterial immune system. Bacteria use CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) as an adaptive immune memory — recording the genetic signatures of viruses that have attacked them and deploying Cas9, a protein that acts as molecular scissors, to cut and destroy the viral DNA if encountered again. Scientists Jennifer Doudna (UC Berkeley) and Emmanuelle Charpentier (Max Planck Institute) realised in 2012 that this system could be reprogrammed to cut any DNA sequence by changing the guide RNA. They were awarded the Nobel Prize in Chemistry in 2020. CRISPR’s revolutionary advantage over previous gene editing tools: it is fast, cheap, precise, and programmable. What previously required years and millions of dollars in laboratory work can now be done in weeks and thousands. The result: an explosion of research, clinical trials, and now approved therapies that has transformed the possibility frontier of medicine. CRISPR can cut, delete, insert, correct, or silence any gene in any cell. The question is no longer whether we can edit the genome. It is whether we should, how, for whom, and who decides. |
| 2 | Way 1 — Treating inherited genetic diseases through somatic cell editing (Casgevy and beyond): Casgevy (exagamglogene autotemcel), approved by the FDA in December 2023, is the world’s first CRISPR-based medicine. It treats sickle cell disease — a debilitating, life-shortening inherited blood disorder caused by a single mutation in the HBB gene affecting haemoglobin structure — and beta-thalassemia. The therapy extracts the patient’s own blood stem cells, uses CRISPR/Cas9 to reactivate the production of fetal haemoglobin (HbF — which is naturally produced before birth and prevents sickling), then reinfuses the edited cells. The result: clinical trials showed that 93.5% of sickle cell patients treated with Casgevy were free from vaso-occlusive crises for at least 12 months. By early 2026, 50 treatment centres across North America, Europe, and the Middle East were actively treating patients. The personalised CRISPR therapy for Baby KJ (NEJM, May 2025) extended this paradigm further: a unique therapy designed for a single patient’s unique mutation in 6 months, correcting a single DNA letter in his liver cells using a base editor delivered in vivo by lipid nanoparticles. |
| 3 | Way 2 — Engineering cancer immunotherapy through CRISPR-enhanced immune cells: Cancer immunotherapy — harnessing the immune system to fight cancer — is one of the most active areas of CRISPR application. CAR-T cell therapy (chimeric antigen receptor T cells) involves extracting a patient’s T cells, genetically engineering them to target cancer cells, and reinfusing them. CRISPR enhances CAR-T therapy in multiple ways: by knocking out genes that suppress T cell activity in the tumour microenvironment (particularly PD-1 and CTLA-4 checkpoint genes); by engineering allogeneic (donor-derived) universal CAR-T cells that do not require patient-specific manufacturing; and by identifying new cancer target genes through CRISPR genome-wide screening. CAR-NK cell therapies, similarly CRISPR-enhanced, are being developed as an alternative with lower toxicity profiles. Multiple clinical trials in haematological cancers (leukaemia, lymphoma) and solid tumours are in Phase I and II stages. The broader implications: CRISPR is moving oncology from chemotherapy — which indiscriminately attacks dividing cells — toward precision molecular targeting. |
| 4 | Way 3 — Germline editing — the heritable rewriting that is banned but has already happened: Germline editing modifies eggs, sperm, or embryos — changes that would be inherited by all future descendants. It represents the most profound and most controversial application of CRISPR. In November 2018, Chinese scientist He Jiankui announced the birth of twin girls from CRISPR-edited embryos — the world’s first germline-edited humans. He claimed to have disabled the CCR5 gene to confer HIV resistance, despite the availability of far simpler preventive alternatives and the medically unnecessary nature of the intervention. The international scientific response was unanimous condemnation. He was imprisoned for three years. But the precedent has been set: germline editing of human beings has already happened. The genie cannot be put back in the bottle. What remains is governance — the question of under what conditions, if any, germline editing of human beings would be ethically permissible, and who has the authority to answer that question. |
| 5 | The 3 questions no one is asking — Question 1: Who defines a genetic defect? The most philosophically important question raised by CRISPR is the one that seems the most obvious once asked: who decides which genetic variants are defects that should be corrected? Sickle cell disease and CPS1 deficiency are clearly debilitating conditions with severe consequences for survival and quality of life. The therapy-enhancement boundary seems clear in these cases. But the boundary becomes genuinely contested when applied to: deafness (many in the Deaf community do not regard deafness as a defect but as a different way of being human — and would object to CRISPR being used to prevent it); Down syndrome; autism spectrum conditions; genetic variants associated with higher rates of certain cancers but not guaranteeing them; and future genetic associations with psychological traits, personality dimensions, or cognitive patterns. The history of the 20th century — including the eugenics programmes that led to forced sterilisation and genocide — is the necessary context for evaluating who has the authority to define genetic normalcy and its deviations. |
| 6 | Question 2: Who can afford it? Casgevy costs approximately $2.2 million per patient — making it one of the most expensive medicines in history. This price is not arbitrary: the development costs of CRISPR therapies are enormous, the manufacturing is complex, and the commercial pathway for therapies targeting rare diseases is narrow. But the consequences of this pricing are ethically significant. Sickle cell disease disproportionately affects people of African, South Asian, Middle Eastern, and Mediterranean descent — communities that are, on average, less wealthy than the populations of the high-income countries where Casgevy is available. India has approximately 20 million sickle cell disease carriers — among the world’s highest burdens. Access to Casgevy in India is currently not a realistic pathway for any patient. The promise of CRISPR as a democratic technology that could eliminate heritable diseases is contradicted by a pricing structure that makes it available only to those whose governments can pay $2.2 million per patient, or whose private insurance provides coverage. If CRISPR’s therapeutic applications remain accessible only to the wealthy, it will not eliminate genetic inequality — it will amplify it. |
| 7 | Question 3: What does the Indian philosophical tradition say about rewriting the human body? The Indian philosophical tradition offers a framework for the CRISPR conversation that Western bioethics — centred primarily on individual autonomy, risk-benefit calculation, and procedural consent — does not provide. The Panchamahabhuta understanding of the body as a sacred construction from five universal elements (Prithvi/earth, Jal/water, Agni/fire, Vayu/air, Akasha/space) implies that the body is not merely a biological machine to be optimised but a localised expression of universal principles — sacred matter that carries responsibility beyond the individual. The concept of Karma-Sanskara — the inscribed record of actions that shapes future manifestation — raises a specific question about germline editing: if future human beings will inherit a genome that has been deliberately modified by choices made before their birth, what does that imply for their Karma? Their Dharma? Their freedom to be what their own nature intended? The Yoga Sutras’ Prakriti (matter, including the genome) and Purusha (consciousness, the observer) distinction raises the question: can editing Prakriti permanently change the relationship between a body and the consciousness that inhabits it? These are not rhetorical questions. They are genuine philosophical challenges that the speed of CRISPR development has not waited for anyone to answer. |
What CRISPR Actually Is — The Molecular Scissors That Won the Nobel Prize
CRISPR-Cas9 did not originate in a laboratory. It originated in bacteria — the oldest and most resilient forms of life on earth. Bacteria have been fighting viruses for billions of years, and over that time they evolved an adaptive immune system: when a virus attacks a bacterium, the bacterium can capture a fragment of the virus’s genetic code and store it in a special region of its own genome called the CRISPR array. If the same virus attacks again, the bacterium transcribes the stored sequence into a guide RNA that leads the Cas9 protein — a molecular cutting enzyme — directly to the viral DNA and slices it apart, neutralising the infection.
In 2012, Jennifer Doudna at UC Berkeley and Emmanuelle Charpentier at the Max Planck Institute realised that this bacterial immune system could be reprogrammed. Change the guide RNA — the sequence that tells Cas9 where to cut — and you can direct the molecular scissors to cut any DNA sequence in any organism. They published their proof of concept in Science (Doudna and Charpentier, Science 2012, DOI: 10.1126/science.1225829). They received the Nobel Prize in Chemistry in 2020.
The revolutionary implication was immediately recognised by the scientific community: CRISPR is fast, cheap, precise, and programmable. Previous gene-editing tools — zinc-finger nucleases and TALENs — could achieve similar effects but required laborious protein engineering for each new target and cost tens of thousands of dollars per experiment. CRISPR requires only changing the guide RNA sequence — a straightforward molecular biology procedure costing a few hundred dollars. The result was an explosion of research that has produced, in little over a decade, the world’s first approved gene therapy using this technology and hundreds of active clinical trials across disease areas that were previously considered incurable.
How CRISPR Works — The Three-Step Mechanism
Step 1: A guide RNA (gRNA) is designed to match the target DNA sequence — the specific stretch of genome to be edited. This is the programmable element: changing the gRNA sequence changes the target. Step 2: The gRNA-Cas9 complex searches through the genome until it finds the matching sequence. Cas9 makes a double-strand cut — breaking both strands of the DNA double helix — at the precise location. Step 3: The cell’s own DNA repair mechanisms activate. Scientists exploit two of these mechanisms: non-homologous end joining (NHEJ), which typically disrupts the cut gene (useful for knocking out a gene); and homology-directed repair (HDR), which uses a provided template to make a precise edit (useful for correcting a mutation or inserting a new sequence).
Next-generation tools build on this mechanism with greater precision. Base editing — the technology used for Baby KJ’s personalised therapy and for Beam Therapeutics’ sickle cell trial — makes single-letter DNA corrections without creating a double-strand break, significantly reducing off-target effects and safety risks. Prime editing, described by its developers as the ‘search-and-replace’ of the genome, can make any point mutation, insertion, or deletion without a double-strand break or a donor DNA template. These advances are progressively reducing the risks while expanding the precision and range of what CRISPR-based approaches can achieve.
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CRISPR did not give us the power to edit the genome. Nature gave bacteria that power three billion years ago. We merely borrowed it — and must now decide whether we are wise enough to use it.
— Dr. Narayan Rout | TheQuestSage.com
Way 1: Treating Inherited Genetic Diseases — From Casgevy to Personalised Medicine
The FDA approval of Casgevy in December 2023 is correctly described as a milestone in medical history. For sickle cell disease — a genetic disorder affecting approximately 100,000 Americans and 20 million carriers in India alone — the therapeutic options before Casgevy were pain management, blood transfusions, and for a minority of patients, bone marrow transplantation. A one-time treatment that eliminates the debilitating crises of sickle cell disease by editing the patient’s own stem cells represents something genuinely new in medicine: a cure for a genetic disease at the molecular level.
The mechanism is elegant. Sickle cell disease is caused by a mutation in the HBB gene that produces an abnormal haemoglobin, causing red blood cells to collapse into a rigid sickle shape that blocks blood vessels and causes severe pain crises, organ damage, and shortened lifespan. Casgevy does not correct the HBB mutation directly. Instead, it uses CRISPR to reactivate the production of fetal haemoglobin (HbF) — the form of haemoglobin the body produces before birth, which does not sickle, and which is normally silenced after birth. With HbF restored to high levels, the sickling is prevented even though the HBB mutation remains.
Baby KJ — The Most Personal Medicine in History
The case of KJ Muldoon, published in the New England Journal of Medicine in May 2025, represents a different paradigm shift. Casgevy is a standardised therapy — the same product for all eligible patients with sickle cell disease. Baby KJ’s therapy was designed for a single patient’s unique mutation in the CPS1 gene — a gene encoding a liver enzyme essential for ammonia metabolism. The mutation was specific to KJ; no pre-existing therapy existed for it.
The team at CHOP and Penn, led by Kiran Musunuru and Rebecca Ahrens-Nicklas, used a base editor — a next-generation CRISPR tool that corrects a single DNA letter without creating a double-strand break — to correct the specific mutation in KJ’s CPS1 gene. The editor was delivered to liver cells via lipid nanoparticles (the same delivery technology used in COVID-19 mRNA vaccines) administered intravenously — no extraction of cells, no bone marrow conditioning, no transplantation. The therapy was designed, tested in cell models and mice, manufactured, and administered in just six months.
The implications of this case extend far beyond CPS1 deficiency. The technology platform is reusable. The base editor components are standardised; only the guide RNA sequence — which directs the editor to the specific target — needs to be customised per patient. KJ’s case demonstrates that CRISPR can deliver personalised genetic medicine for rare diseases — the 7,000+ rare genetic diseases that collectively affect 300 million people globally, most of which currently have no approved therapy — in clinically relevant timeframes and without requiring hospitalisation for cell extraction and transplantation.
For the genetics and neuroscience of consciousness and the broader questions about what DNA and the genome mean for human identity, see The Genetics of Consciousness: 5 Things DNA and Darshan Both Say About Who We Are (TheQuestSage.com).
Way 2: Engineering Cancer Immunotherapy — Teaching the Immune System to Fight
Cancer is not one disease. It is hundreds of diseases, each defined by a specific pattern of genetic mutations in a specific cell type, with a unique combination of surface markers, growth drivers, and immune evasion mechanisms. The promise of CRISPR for cancer is the promise of molecular precision: the ability to engineer therapies targeted to the specific genetic characteristics of each patient’s tumour rather than using chemotherapy that indiscriminately attacks all rapidly dividing cells.
CRISPR and CAR-T Cell Therapy
CAR-T cell therapy — chimeric antigen receptor T-cell therapy — is one of the most significant immunotherapy advances of the past decade. It involves extracting the patient’s T cells, engineering them in the laboratory to express a receptor that recognises cancer cells, and reinfusing them. CAR-T therapies have produced remarkable remissions in some haematological cancers — leukaemia and lymphoma — that had been refractory to all other treatments.
CRISPR enhances CAR-T therapy through multiple mechanisms. Knocking out PD-1 and CTLA-4 checkpoint genes in engineered T cells prevents tumour microenvironment suppression — removing the molecular brakes that tumours exploit to escape immune attack. Engineering allogeneic (donor-derived, universal) CAR-T cells through CRISPR-mediated HLA matching elimination allows off-the-shelf manufacturing rather than patient-specific production, making the therapy faster and potentially cheaper. Genome-wide CRISPR screening — systematically disrupting every gene in cancer cells to identify which genes are essential for survival — identifies new therapeutic targets that conventional approaches would have missed.
Multiple CRISPR-enhanced CAR-T and CAR-NK cell trials are in Phase I and II stages for haematological malignancies. The extension to solid tumours — which have been far more resistant to immunotherapy — is an active research frontier. The 239 active gene-editing clinical trials documented by CRISPR Medicine News in 2024 span this landscape.
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Chemotherapy asks: what kills cells? CRISPR immunotherapy asks: what makes this particular cancer cell different from this particular patient’s healthy cells? That is the difference between a blunt instrument and a scalpel.
— Dr. Narayan Rout | TheQuestSage.com
Way 3: Germline Editing — The Heritable Rewriting That Is Banned but Has Already Happened
The He Jiankui affair is the most important single event in the history of CRISPR ethics — not because He Jiankui succeeded in a scientific sense (the medical justification for his intervention was essentially non-existent) but because he demonstrated irrevocably that human germline editing is technically possible and that the governance frameworks in place in 2018 were insufficient to prevent a determined scientist from using it outside any ethical oversight structure.
He Jiankui edited the CCR5 gene in embryos that were subsequently implanted in women, resulting in the births of twin girls — Lulu and Nana — and later a third child. CCR5 is a co-receptor that HIV uses to enter T cells; people with a naturally occurring CCR5 deletion variant are resistant to certain strains of HIV. He claimed his edit was medically justified because the children’s fathers were HIV-positive — despite the fact that sperm-washing, a well-established and much simpler technique, reduces HIV transmission risk to less than 1%. The medical justification for creating heritable changes in three human genomes and all their future descendants was, in the judgment of the international scientific community, essentially zero.
The Governance Response — And Its Limits
The international response was swift and near-unanimous: condemnation. The WHO’s Expert Advisory Committee stated in 2019 that it was irresponsible for anyone to proceed with human germline genome editing. Eighteen leading scientists called for a moratorium. The Third International Summit on Human Genome Editing reaffirmed in 2023 that human germline editing is not acceptable since safety, ethical, and governance standards have not been met. Germline editing is banned in China, India, Brazil, Singapore, and many other countries.
But the governance response has a structural limitation that is rarely acknowledged: international scientific consensus is not the same as international law. There is no treaty, no binding international legal instrument, that prohibits human germline editing. What exists is a patchwork of national regulations with varying levels of enforcement — and the demonstration, by He Jiankui, that a scientist motivated to proceed and working in a jurisdiction with inadequate oversight can do so without prior detection. The genie has been released. The governance question is not whether it can be put back. It is how to ensure that the next application of germline editing — if and when it occurs — does so within a framework of scientific rigour, ethical review, public deliberation, and equitable access rather than the unilateral action of a single scientist with access to CRISPR equipment.
The 3 Questions No One Is Asking
Question 1: Who Defines a Genetic Defect?
The therapy-enhancement distinction that underpins current CRISPR ethics assumes a clear answer to the question: which genetic variants are defects that should be corrected, and which are part of the normal range of human genetic diversity? For sickle cell disease, the answer seems clear — a mutation that causes severe pain crises, organ damage, and shortened life is a defect. For CPS1 deficiency, which threatens life in infancy, the answer is equally clear.
But the clarity dissolves rapidly as the range of potential CRISPR applications expands. In 2023, the Deaf community and disability rights advocates raised significant objections to proposals to use CRISPR to prevent genetic deafness — pointing out that the Deaf community does not widely regard deafness as a defect but as a different way of being human with its own language, culture, and community. The framing of CRISPR as a tool to ‘fix’ deafness assumes a definition of normal hearing that the Deaf community has specifically rejected. Similar arguments apply to certain autism spectrum conditions, to Down syndrome advocacy communities, and potentially — as genetic associations with psychological traits become better understood — to genetic variants associated with introversion, with unconventional cognition, or with psychological states that contemporary society labels as disorders.
The eugenic impulse — the drive to eliminate what is defined as genetically inferior in order to produce a ‘better’ human stock — does not require a formal government programme. It requires only a technology that can eliminate genetic variants, a definition of which variants are undesirable, and a social context in which genetic testing and editing become normal features of reproduction. The current trajectory of CRISPR development, without explicit governance frameworks that address the definition-of-defect problem, risks reproducing the logic of eugenics through private medical decision-making rather than state compulsion.
Question 2: Who Can Afford It?
The $2.2 million price tag of Casgevy is the most direct statement of the access problem. Sickle cell disease is not a disease of the wealthy. It is a disease disproportionately affecting people of African, South Asian, Middle Eastern, and Mediterranean descent — communities whose global distribution skews toward low and middle-income countries. India’s sickle cell disease burden — approximately 20 million carriers, with approximately 12,000-15,000 affected births annually — is among the world’s highest. The Indian government’s Sickle Cell Anaemia Mission aims to screen and provide basic support but has no realistic pathway to providing $2.2 million therapies at scale.
The access problem is not unique to CRISPR — it characterises pharmaceutical innovation broadly. But it is particularly acute for CRISPR because the technology’s promise is explicitly universal: CRISPR’s advocates describe it as a tool that will eliminate heritable diseases. If that elimination is available only to patients in countries wealthy enough to reimburse a $2.2 million therapy, the promise is not universal — it is a promise to the wealthy with the implication that the heritable disease burden will become increasingly concentrated in the populations that cannot afford the cure. This would represent a new dimension of global health inequity built on a technology that was initially celebrated for its democratic accessibility.
Question 3: What Does the Indian Philosophical Tradition Say?
The dominant framework for CRISPR ethics is Western bioethics: individual autonomy, informed consent, risk-benefit analysis, and procedural governance. This framework has produced important safeguards and will continue to be necessary. But it is insufficient — because it does not have the philosophical vocabulary to address the most profound question raised by germline editing: the question of the authority to make permanent, heritable changes to the genetic substrate of future human beings who cannot consent and whose existence depends on the decisions being made.
अहं ब्रह्मास्मि — तत् त्वम् असि |
– Brihadaranyaka Upanishad 1.4.10 and Chandogya Upanishad 6.8.7
I am Brahman. That thou art. The individual self is not separate from the universal. The genome is not merely individual property — it is a localised expression of universal intelligence.
The Panchamahabhuta framework — the understanding that the body is constituted from five universal elements (earth, water, fire, air, and space) that are not the individual’s property but expressions of universal principles — raises a question that Western bioethics does not: if the genome is a localised expression of universal intelligence operating through the five elements, what is the ethical standing of the decision to permanently alter it? Who has the authority to permanently rewrite what the universe, through billions of years of evolutionary intelligence, has written?
The Karma-Sanskara framework adds a further dimension. Karma — the law of action and consequence — and Sanskara — the inscribed impressions that shape future manifestation — together describe a principle in which what has been done becomes the substrate of what comes next. Germline editing creates a Sanskara in the genome of future generations — an inscription made before their birth that they cannot alter and did not choose. The ethical question of consent, which Western bioethics addresses procedurally, is addressed philosophically in the Indian tradition through the concept of Dharma: the rightness of action in accordance with the nature of things. Is it Dharmic to inscribe a choice into the genome of beings who will not exist until after the choice has been made?
These are not questions with simple answers. They are questions whose depth the Western bioethics framework does not fully capture — and whose exploration the speed of CRISPR development has not waited for.
The Quest Sage Insight
want to offer a perspective on the CRISPR conversation that goes beyond the science and the ethics as usually framed.
We are in the early decades of humanity’s first genuinely transformative capability to edit its own biological code. The human genome — three billion base pairs, encoding approximately 20,000 genes, shaped by four billion years of evolutionary history — is not primarily a medical record of defects to be corrected. It is, in the deepest sense, the biological expression of what the universe has learned about how to build a conscious being. Every genetic variant that has persisted in the human population has persisted because, at some point in some environment, it conferred some advantage or at minimum did not impose sufficient disadvantage to be eliminated. The HbS mutation that causes sickle cell disease persists in malaria-endemic populations because the heterozygous carrier state — one copy of the mutation — confers significant malaria resistance. The genetic variants associated with certain psychological intensities may confer creative or perceptual capacities alongside their vulnerabilities.
None of this means that CRISPR should not be used to treat sickle cell disease. Baby KJ’s successful personalised therapy is one of the most beautiful stories in the history of medicine — a team of dedicated scientists and clinicians devoting themselves to saving one child’s life with the most sophisticated molecular tools humanity has ever produced. The goodness of that act is not diminished by the complexity of the questions the same technology raises in other contexts.
What I am suggesting is that the Vedic framework’s understanding of the body as sacred matter — not sacred in a superstitious sense but in the sense of being the product of intelligence that exceeds individual human comprehension — offers a useful caution against the hubris that CRISPR’s power invites. The ability to edit the genome does not automatically confer the wisdom to know which edits are improvements. The track record of human technology in other domains — nuclear energy, industrial agriculture, social media algorithms — suggests that the capacity to do something and the wisdom to know whether it should be done are not the same capacity, and do not develop at the same pace.
The three questions no one is asking — who defines a defect, who can afford the cure, and what civilisational frameworks provide the ethical architecture — are not obstacles to CRISPR’s therapeutic development. They are the conditions that would make that development genuinely beneficial rather than merely technically impressive. The difference between medicine and enhancement, between equity and privilege, between wisdom and power — these are the questions that the speed of CRISPR development must somehow find time for.
✅ 3 Key Outcomes
1. CRISPR is a present technology with approved therapies, not a future speculation. Casgevy, FDA-approved in December 2023, is the world’s first CRISPR-based medicine — treating sickle cell disease and beta-thalassemia by editing patients’ blood stem cells to reactivate fetal haemoglobin production. Baby KJ received the world’s first personalised CRISPR base-editing therapy in May 2025 (NEJM, Musunuru et al., DOI: 10.1056/NEJMoa2504747) — designed for his unique CPS1 gene mutation in 6 months, delivered in vivo via lipid nanoparticles. 239 active gene-editing clinical trials in 2024 span cancer immunotherapy, hereditary disease, and chronic viral infections. Next-generation base editing and prime editing reduce off-target risks while expanding precision.
2. The most important ethical distinction is somatic versus germline editing. Somatic editing modifies non-reproductive cells — affects only the patient, not heritable. Casgevy and Baby KJ’s therapy are both somatic. Broadly supported by the scientific community. Germline editing modifies eggs, sperm, or embryos — heritable changes to all future descendants. Currently banned or under moratorium in most countries including India. He Jiankui’s 2018 creation of the world’s first germline-edited human births resulted in his imprisonment and near-universal condemnation. The Third International Summit reaffirmed in 2023 that clinical germline editing is not acceptable. The three questions CRISPR raises that are not being adequately addressed: who defines a genetic defect, who can afford the $2.2 million cure, and what philosophical frameworks provide the ethical architecture for editing the human genome.
3. The Indian philosophical tradition offers depth to the CRISPR conversation that Western bioethics lacks. The Panchamahabhuta understanding of the body as sacred matter — a localised expression of five universal elements — raises the question of the authority to permanently alter what the universe has written through evolutionary intelligence. The Karma-Sanskara framework raises the question of heritable editing: is it Dharmic to inscribe a choice into the genome of beings who cannot consent because they do not yet exist? The Advaita identification of the individual with the universal — Aham Brahmasmi — frames the genome not as individual biological property to be optimised but as a localised expression of universal intelligence whose alteration carries responsibility beyond the individual. India has 20 million sickle cell disease carriers and is banned from germline editing. India’s specific disease burden and civilisational resources make it one of the most important voices in the global CRISPR governance conversation — a voice it has not yet fully deployed.
What You Can Do With This
- Understand the somatic versus germline distinction before forming an opinion on CRISPR ethics. Somatic editing for serious genetic disease — treating a patient’s own cells, affecting only that patient — is broadly supported by the scientific community and has produced the first approved gene therapy. Germline editing — making heritable changes to embryos — is currently banned or under moratorium in most countries including India. These are not the same thing. Most CRISPR development happening now is somatic. Most CRISPR concern expressed in popular discourse conflates somatic therapy with germline enhancement. The distinction matters.
- For Indians with genetic disease burden in the family — explore what is coming. India has significant sickle cell disease, thalassemia, and other monogenic disease burdens. The CRISPR therapies that are FDA-approved or in clinical trials now may become accessible in India in the next decade. The Danaher-IGI Beacon for CRISPR Cures explicitly focuses on affordable manufacturing and global accessibility. The Indian government’s Sickle Cell Mission is a relevant policy context. Understanding what is coming is the prerequisite for advocating for equitable access.
- Engage with the philosophical questions — not just the scientific ones. The CRISPR conversation in India tends to be dominated by either uncritical enthusiasm for the technology or blanket rejection on religious grounds. Both responses miss the genuine complexity. The Panchamahabhuta and Karma-Sanskara frameworks are not obstacles to understanding CRISPR — they are resources for thinking about it with more depth than the Western bioethics framework alone provides. What does it mean to be the kind of being whose genome can be edited? That is a question worth sitting with.
- Follow the Baby KJ story as a benchmark for personalised medicine. The technology platform demonstrated by KJ’s case — base editing delivered by lipid nanoparticles, designed to a specific patient’s unique mutation in 6 months — is a platform with implications far beyond CPS1 deficiency. The team at CHOP and Penn explicitly stated that this platform can be extended to hundreds of rare diseases. Following the scientific and regulatory development of this platform over the next 5-10 years will tell you more about the future of medicine than almost any other single story to watch.
Conclusion: The Most Powerful Tool Medicine Has Ever Had — And the Wisdom It Still Needs
Three things are happening simultaneously in CRISPR medicine right now. Casgevy is treating sickle cell disease patients at 50 active centres across three continents, offering clinical cure to people who previously had no option beyond managing their pain. Baby KJ is home after the first personalised CRISPR therapy, demonstrating that a technology platform for rare genetic diseases can be designed and delivered in months rather than decades. And 239 active clinical trials are exploring applications from cancer immunotherapy to cholesterol reduction to chronic hepatitis B.
At the same time: Casgevy costs $2.2 million per patient, placing it beyond reach for the vast majority of the world’s sickle cell disease population. He Jiankui’s three germline-edited children are growing up somewhere in China with changes to their DNA — and their descendants’ DNA — that no governance framework prevented and that no international agreement can reverse. And the question of who defines genetic normalcy and its deviations — the foundational question that determines how far CRISPR editing should go — has no agreed answer.
The technology is not waiting for the ethics. It never does. What the Indian philosophical tradition offers — the Panchamahabhuta’s understanding of the body as sacred matter, the Karma-Sanskara framework for evaluating the authority to make permanent inscriptions in future genomes, the Advaita insight that the individual genome is a localised expression of universal intelligence — is not a reason to stop CRISPR development. It is a reason to develop it with the depth of reflection that its power demands.
We are rewriting the human genome. The question is whether we are rewriting it wisely.
🪞 3 Self-Reflection Questions
Q1. The Deaf community argues that deafness is not a defect but a different way of being human, with its own language, culture, and community — and that using CRISPR to prevent genetic deafness is not medical treatment but cultural elimination. What is your response to this argument? Where do you draw the line between a genetic variant that should be corrected and one that is part of human diversity?
Q2. Baby KJ’s personalised therapy — designed for a single patient’s unique mutation in 6 months at a cost currently accessible only in a major research hospital in a wealthy country — demonstrates what CRISPR can do in principle. What would it mean for this capability to be genuinely globally accessible? What would need to change in the economic structure of pharmaceutical development, in global health governance, and in India’s research infrastructure for Indian patients with rare genetic diseases to have realistic access to personalised genetic medicine within a generation?
Q3. The Upanishad says Aham Brahmasmi — I am Brahman. The genome is a localised expression of universal intelligence. If you were the parent of a child with a heritable genetic disease, and a safe and effective CRISPR germline therapy existed that would correct the mutation not just in your child but in all their future descendants — permanently eliminating the disease from your family line forever — would you use it? What does your answer reveal about your relationship to the Panchamahabhuta understanding of the body?
Frequently Asked Questions: Gene Editing and CRISPR
Q1. What is CRISPR and how does gene editing work?
CRISPR-Cas9 is a gene-editing technology derived from the bacterial immune system. Bacteria evolved CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) as an adaptive immune memory — storing fragments of viral genetic code and using the Cas9 protein as molecular scissors to cut and destroy viral DNA if encountered again. In 2012, Jennifer Doudna and Emmanuelle Charpentier reprogrammed this system to cut any target DNA sequence by changing the guide RNA (a short RNA molecule that leads Cas9 to the target). They were awarded the Nobel Prize in Chemistry in 2020. CRISPR works in three steps: (1) a guide RNA matching the target DNA sequence is designed; (2) the guide RNA-Cas9 complex finds and cuts the target DNA at the precise location; (3) the cell’s DNA repair mechanisms activate — either disrupting the cut gene or, if a repair template is provided, making a precise edit. Next-generation tools include base editing (correcting single DNA letters without a double-strand cut) and prime editing (a search-and-replace mechanism). CRISPR is faster, cheaper, more precise, and more programmable than previous gene-editing technologies, making it accessible to research laboratories globally and enabling the current explosion of clinical applications.
Q2. What is Casgevy and why is it significant?
Casgevy (exagamglogene autotemcel) is the world’s first FDA-approved medicine using CRISPR/Cas9 technology, approved in December 2023 for sickle cell disease in patients aged 12 and older, and subsequently approved for beta-thalassemia. It was developed by Vertex Pharmaceuticals and CRISPR Therapeutics. Sickle cell disease is caused by a mutation in the HBB gene that produces abnormal haemoglobin, causing red blood cells to collapse into a rigid sickle shape that blocks blood vessels and causes severe pain crises, organ damage, and shortened lifespan. Casgevy works by extracting the patient’s own haematopoietic (blood) stem cells, using CRISPR/Cas9 to reactivate the production of fetal haemoglobin (HbF — which does not sickle and which the body normally stops producing after birth), then reinfusing the edited cells. Clinical trials showed 93.5% of sickle cell patients treated were free from vaso-occlusive crises for at least 12 months. Casgevy is significant because it demonstrates that CRISPR-based medicine works at the clinical level — that gene editing technology developed in academic laboratories can be translated into a safe and effective treatment for real patients with serious genetic disease. The approvals by the FDA, EMA, UK MHRA, Health Canada, and other agencies confirm that CRISPR medicine has met regulatory standards for safety and efficacy. The main limitation: its price of approximately $2.2 million per patient limits access to wealthy countries and well-resourced health systems.
Q3. What happened with Baby KJ and why is it a breakthrough?
Baby KJ is an infant born with severe carbamoyl phosphate synthetase 1 (CPS1) deficiency — a rare genetic metabolic disease in which a defective liver enzyme cannot process protein properly, causing toxic ammonia to accumulate in the blood. Only about half the babies with this condition survive long enough to receive a liver transplant. The conventional treatment pathway — liver transplant — requires waiting for a compatible donor organ. A team led by Kiran Musunuru and Rebecca Ahrens-Nicklas at Children’s Hospital of Philadelphia (CHOP) and the University of Pennsylvania designed, manufactured, and administered a personalised CRISPR base-editing therapy targeting the specific mutation in KJ’s CPS1 gene in just six months. The therapy was delivered intravenously via lipid nanoparticles — no cell extraction, no bone marrow transplant, no hospitalisation for cell manufacturing. Within seven weeks of the first infusion, KJ was tolerating more dietary protein and on half his starting medication dose. He was discharged after 307 days in hospital. The case was published in the New England Journal of Medicine on May 15, 2025 (Musunuru et al., DOI: 10.1056/NEJMoa2504747). The breakthrough: the technology platform used for KJ — base editing delivered by lipid nanoparticles, customised to a specific patient’s unique mutation in months — is reusable. The guide RNA sequence needs changing per patient; the rest of the platform is standardised. This creates a pathway to personalised genetic medicine for the 7,000+ rare genetic diseases that collectively affect 300 million people globally and most of which have no approved therapy.
Q4. What is the difference between somatic and germline editing?
This is the most important distinction in CRISPR ethics. Somatic cell editing modifies non-reproductive cells — blood cells, liver cells, muscle cells — that affect only the individual patient being treated. Changes made to somatic cells are not passed to the patient’s children. Casgevy is somatic editing: it modifies the patient’s blood stem cells but does not change the germline. Baby KJ’s therapy is somatic editing: it corrected his liver cells specifically targeted to his non-reproductive tissue. Somatic editing for serious genetic disease is broadly supported by the scientific and medical community. Germline editing modifies eggs, sperm, or embryos — the reproductive cells that contribute to the genomes of offspring. Any changes to germline cells are heritable: they would be passed to all future descendants, affecting every generation in perpetuity. Germline editing is currently banned or under moratorium in most countries for clinical use, including India, China, Brazil, Singapore, and Uganda. The fundamental ethical distinction: somatic editing respects individual consent (the patient or their guardians can decide); germline editing affects future generations who cannot consent to having their genome permanently altered by decisions made before their birth. He Jiankui violated this principle by editing embryos without adequate ethical oversight, resulting in three children with heritable genetic modifications and his subsequent imprisonment.
Q5. Is CRISPR gene editing regulated in India?
India has banned germline genome editing under its existing biomedical research regulations. The Indian Council of Medical Research (ICMR) and the Department of Biotechnology (DBT) Guidelines for Stem Cell Research (2017, revised 2021) prohibit the clinical use of genome-edited embryos — consistent with the positions of China, Brazil, Singapore, Uganda, and many other countries. Somatic cell gene editing research is permitted under appropriate ethical oversight. India has significant genetic disease burdens that CRISPR therapeutic development is directly relevant to: approximately 20 million sickle cell disease carriers (one of the world’s highest burdens), approximately 10,000 thalassemia-affected births annually, and significant disease burden from founder mutations in specific communities. India’s Sickle Cell Anaemia Mission aims to provide screening and basic support but currently has no realistic pathway to providing CRISPR therapies at $2.2 million per patient at scale. India has world-class biotechnology research capacity — IITs, IISc, NCBS, CCMB, and other institutions — that could contribute to developing more affordable CRISPR manufacturing approaches, potentially making these therapies accessible to the Indian population at a scale consistent with the country’s disease burden and resources.
Q6. What are the risks of CRISPR gene editing?
CRISPR gene editing carries several categories of risk that researchers and regulators are actively working to understand and mitigate. Off-target effects: CRISPR can sometimes cut at unintended locations in the genome where the guide RNA sequence is similar but not identical to the target. Unintended cuts could disrupt normal gene function or, theoretically, activate oncogenes (cancer-causing genes). Next-generation base editing and prime editing technologies significantly reduce double-strand breaks and thereby reduce off-target effects. Delivery challenges: getting CRISPR components into the right cells in the right amounts is technically demanding, particularly for in vivo (inside the body) editing. Lipid nanoparticles — the same technology used in COVID-19 mRNA vaccines — are the most clinically advanced delivery system. Immune response: the body may recognise CRISPR components as foreign and mount an immune response that limits effectiveness or causes adverse effects. Long-term safety: as an approved medicine, Casgevy’s long-term effects are being monitored in ongoing trials. Most current clinical experience is limited to several years. For germline editing specifically: any off-target effects or unintended consequences would be heritable — passed to all future descendants — and irreversible. This is the primary reason germline editing is held to a much higher safety standard than somatic editing and is currently not permitted for clinical use in most countries.
Q7. What does Indian philosophy say about gene editing?
The Indian philosophical tradition offers several frameworks that are directly relevant to CRISPR ethics, though they do not provide simple verdicts. The Panchamahabhuta framework understands the body as constituted from five universal elements — Prithvi (earth), Jal (water), Agni (fire), Vayu (air), and Akasha (space) — that are not the individual’s private property but expressions of universal principles. This understanding implies that the body carries responsibility beyond the individual — that editing it, especially heritably, involves a relationship with the universal order that individual autonomy and risk-benefit analysis do not fully capture. The Karma-Sanskara concept — the inscribed record of actions that shapes future manifestation — raises a specific question about germline editing: changes made to the germline create a Sanskara in the genomes of future generations, an inscription made before their birth that they cannot alter and did not choose. The Advaita identification of Atman with Brahman — the individual with the universal — implies that the genome is not merely individual biological material to be optimised but a localised expression of universal intelligence operating through the specific conditions of a particular life. The Yoga Sutras’ Prakriti-Purusha distinction — between matter (including the genome) and consciousness (the observer) — raises the question of whether editing Prakriti can affect Purusha, and what the relationship between the two implies for the ethics of permanent biological alteration. These frameworks do not prohibit CRISPR therapy for serious disease. They provide depth to the conversation that the Western bioethics framework alone does not provide — particularly for the germline editing question.
📖 How to Cite This Article
Rout, N. (2026). Gene Editing and CRISPR: 3 Ways We Are Rewriting the Human Genome Right Now — And the 3 Questions No One Is Asking . TheQuestSage Research Series, TQS-2026-112. https://doi.org/10.5281/zenodo.20627207
License: CC BY 4.0 · Publisher: TheQuestSage.com · ORCID: 0009-0009-3505-5478
References and Sources
1. Doudna, J.A., & Charpentier, E. (2012). A programmable dual-RNA–guided DNA endonuclease in adaptive bacterial immunity. Science, 337(6096), 816–821. DOI: 10.1126/science.1225829. Nobel Prize in Chemistry 2020.
2. FDA. (2023, December 8). FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease. FDA Press Announcement. Casgevy: first FDA-approved CRISPR/Cas9 therapy; CRISPR/Cas9 cuts DNA at targeted areas; edited stem cells increase fetal haemoglobin. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease
3. CRISPR Therapeutics IR. (2024, March 30). Vertex and CRISPR Therapeutics Announce US FDA Approval of CASGEVY for Sickle Cell Disease. ~16,000 eligible patients 12+; US $200 million milestone payment; 50 active treatment sites by early 2026. https://ir.crisprtx.com/news-releases/news-release-details/vertex-and-crispr-therapeutics-announce-us-fda-approval
4. Musunuru, K., Ahrens-Nicklas, R., et al. (2025, May 15). Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease. New England Journal of Medicine. DOI: 10.1056/NEJMoa2504747. Baby KJ; CPS1 deficiency; base editing; lipid nanoparticle delivery; 6-month development; first personalised CRISPR therapy; CHOP/Penn.
5. IGI. (2026, March 23). CRISPR Clinical Trials: A 2025 Update. Innovative Genomics Institute. Casgevy as first CRISPR medicine; Beam Therapeutics BEACON trial base editing; 50 active Casgevy sites; Baby KJ first personalised CRISPR therapy. https://innovativegenomics.org/news/crispr-clinical-trials-2025/
6. NIH. (2025, May 23). Infant with rare, incurable disease is first to successfully receive personalized gene therapy treatment. CHOP and Penn team; Musunuru and Ahrens-Nicklas; CPS1 correction; LNP delivery; 307 days in hospital; discharged June 2025. https://www.nih.gov/news-events/news-releases/infant-rare-incurable-disease-first-successfully-receive-personalized-gene-therapy-treatment
7. Inside Precision Medicine. (2025, August 15). First Personalized CRISPR Gene Editing Therapy Patient Baby KJ Discharged. Danaher-IGI Beacon for CRISPR Cures; scalable delivery; affordable manufacturing; global accessibility goal. https://www.insideprecisionmedicine.com/topics/precision-medicine/first-personalized-crispr-gene-editing-therapy-patient-baby-kj-discharged/
8. CRISPR Medicine News. (2024, December). CRISPR Medicine in 2024: A Recap. 239 active gene-editing trials; Casgevy approvals (UK, FDA, EMA, Saudi Arabia, Canada); hereditary amyloidosis and hereditary angioedema Phase 3 trials; TUNE-401 for hepatitis B; next-generation delivery systems. https://crisprmedicinenews.com/news/crispr-medicine-in-2024-a-recap/
9. Pavani, G., & Guiraud, S. (2024, May 8). CRISPR-Based Gene Therapies: From Preclinical to Clinical Treatments. Cells, 13(10), 800. DOI: 10.3390/cells13100800. PMC: PMC11119143. CRISPR modification of haematopoietic stem cells; blood disorders; cancer; off-target effects; delivery systems.
10. PMC. (2025). Gene Editing: Developments, Ethical Considerations, and Future Directions. PMC11759082. He Jiankui timeline; moratorium calls; NIH position; Third International Summit 2023 reaffirmation; somatic vs germline distinction.
11. IntechOpen. (2025, September 30). Editing the Future: Ethical Challenges of Therapeutic Use, Germline Modification and Human Enhancement with CRISPR. Off-target effects; informed consent; designer babies; germline bans — Brazil, China, India, Singapore, Uganda; enhancement vs correction debate.
12. IGI. (2025, January 21). CRISPR & Ethics. He Jiankui affair; CCR5 edit; twin girls and third child; international condemnation; three years prison; governance response; enhancement concerns. https://innovativegenomics.org/crisprpedia/crispr-ethics/
13. Springer HEC Forum. (2024, September 20). The Ethics of Human Embryo Editing via CRISPR-Cas9: A Systematic Review. DOI: 10.1007/s10730-024-09538-1. Six major themes: risk/harm; benefit; oversight; informed consent; justice/equity; eugenics.
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15. Asamaka Industries. (2026, January 26). CRISPR and Human Ethics of Gene Editing. Conservative debate on germline; safety and benefit evidence requirements; CHOP/Penn personalised therapy as ethical model; equal accessibility as governance goal.
16. Taittiriya Upanishad, Shiksha Valli; Panchamahabhuta — five elements as constituents of the body. Body as expression of universal principles, not individual property.
17. Brihadaranyaka Upanishad 1.4.10. Aham Brahmasmi — I am Brahman. Individual self as expression of universal consciousness; genome as localised expression of universal intelligence.
18. Chandogya Upanishad 6.8.7. Tat Tvam Asi — That Thou Art. The identity of individual and universal; ethical implications for heritable modification.
19. Yoga Sutras of Patanjali. Prakriti-Purusha distinction. Matter (including the genome) and consciousness (the observer); the relationship between biological substrate and the witnessing awareness that inhabits it.
20. Narayan Rout. Yogic Intelligence vs Artificial Intelligence. BFC Publications, 2025. (The inner intelligence that genome editing cannot replicate — Prajna versus the technical intelligence of Vijnana.)
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Dr. Narayan Rout Author · Independent Researcher · Founder, TheQuestSage.com 🏅 Rabindra Ratna Puraskar Awardee |
Dr. Narayan Rout explores the intersection of science, philosophy, consciousness, health, technology, and human development. His work combines evidence-based research with insights from ancient wisdom traditions to make complex ideas accessible to a global audience.
Education & Experience
PG Diploma PM & IR · BNYT · BE (Electrical) · Diploma Industrial Hygiene
Diploma Psychology · Mindfulness · Nutrition · Gut Health
Indian Air Force Veteran (23 Years) · Senior Technician, BHEL
Research Interests
Consciousness Neuroscience Psychology Human Behaviour Health Sciences Technology Civilisation Studies Indian Philosophy
Publications
110+ Published Research Articles · 50+ DOI Registered Works · Zenodo · CERN · OpenAIRE
📚 Books
🔬 Research & Academic Profiles
Further Reading
P10 The Next Human — Science, Technology & Human Evolution
- The Genetics of Consciousness: 5 Things DNA and Darshan Both Say About Who We Are (TheQuestSage.com) — The Orch OR theory, the hard problem of consciousness, and what the genome encodes beyond biology.
- The Road to Super AI: 3 Scenarios That Keep the World’s Smartest People Awake at Night (TheQuestSage.com) — The parallel convergence of AI and CRISPR as the two technologies most likely to reshape humanity.
- Yogic Intelligence vs Artificial Intelligence (TheQuestSage.com) — The intelligence that cannot be edited into a genome — Prajna and the inner development of consciousness.
- Advaita Vedanta and Modern Science: 5 Remarkable Convergences (TheQuestSage.com) — The Aham Brahmasmi framework that contextualises the authority to edit the genome.
📋 Publication Record
| Series | TheQuestSage Research Series |
| Paper Number | TQS-2026-112 |
| Version | 1.0 |
| Publisher | TheQuestSage.com |
| DOI | 10.5281/zenodo.20627207 |
| ORCID | 0009-0009-3505-5478 |
| Language | English |
| License | CC BY 4.0 — Creative Commons Attribution |
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