A 23-year-old man in the US became the first to be functionally cured of sickle cell disease using Casgevy, a CRISPR-based gene-editing therapy
GS3Economy · S&T · Environment · Security· Biotechnology & health research· Prelims + Mains·
Why in news
A 23-year-old man in the US became the first to be functionally cured of sickle cell disease using Casgevy, a CRISPR-based gene-editing therapy.
Background
Casgevy is a CRISPR-based therapy developed by Vertex Pharmaceuticals and CRISPR Therapeutics. It works by using a patient's own blood-forming stem cells to reactivate fetal haemoglobin production to prevent red blood cells from deforming.
Facts for Prelims
- S&TCRISPR-Cas9: A tool for precise genome editing that allows scientists to 'cut' and 'paste' DNA sequences.
- S&TCasgevy: The first CRISPR-based gene-editing therapy approved for sickle cell disease.
- FactThe patient received edited cells in March 2026 and was discharged in April 2026 with normal haemoglobin levels.
- S&TFetal Haemoglobin: A type of haemoglobin produced during fetal development that is normally silenced after birth.
For Mains
Q. Discuss the potential of CRISPR-based gene-editing technologies in revolutionizing the treatment of hereditary blood disorders and the ethical considerations surrounding human genome editing.
Dimensions to cover in your answer
- Ethical dilemma: Balancing the potential for curative gene therapy against the risks of unintended off-target genetic mutations.
- Accessibility gap: High cost of personalized CRISPR therapies creating a disparity in healthcare access for vulnerable populations.
- Regulatory hurdle: Complexity in establishing safety protocols for permanent germline vs. somatic cell gene modifications.
Keywords: Gene Editing · CRISPR-Cas9 · Biotechnology · Hereditary Disorders · Precision Medicine
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