Context:
• CRISPR gene-editing therapy shows successful early human trial results — targeting ANGPTL3 gene → permanent lowering of LDL (‘bad’ cholesterol) & triglycerides.
Key Highlights:
- Gene Target & Mechanism
• ANGPTL3 gene regulates LDL & triglycerides.
• Therapy edits liver cells — primary organ for lipid metabolism.
• Inspired by natural loss-of-function mutation (1 in ~250 people) → lifelong low lipids without adverse effects. - Trial Results (Phase-1 safety trial, 15 patients)
• Highest dose group → ~50% LDL fall, ~55% triglyceride fall.
• Mild infusion-related side effects.
• 1 death (low dose; advanced CVD) → confirmed unrelated. - Regulatory Path Ahead
• Approved to proceed into long-term follow-up → monitoring up to 15 years.
• Phase-2/3 → efficacy + persistent safety.
Relevant Prelims Points:
• ANGPTL3 → key regulator of LDL + triglycerides.
• CRISPR-Cas9 → precise, cell-specific editing (here in liver hepatocytes).
• Natural ANGPTL3 LOF mutation = benign → proof of concept.
• Statins reduce cholesterol via HMG-CoA reductase inhibition — CRISPR approach = one-time, permanent edit.
Relevant Mains Points:
• CVD → India’s largest NCD burden; high cholesterol = primary modifiable risk.
• This therapy → reduces lifetime drug dependence; supports precision-medicine paradigm.
• Ethical Dimensions → germline vs somatic editing, informed consent, cost-access barriers.
• Way Forward:
– indigenous biomanufacturing + regulatory bioethics frameworks
– gene therapy HTA (health technology assessment) for pricing reimbursement
– large Indian cohort trials (genetic diversity)
UPSC Relevance (GS-wise):
• GS2 – Health policy, regulation, equity
• GS3 – Science & Tech, Biotechnology, NCD burden, Precision medicine