India eyes breakthrough against sickle cell
- India is getting closer to developing a gene therapy for sickle cell disease
Highlights:
- Sickle cell disease is a genetic blood disorder with a high prevalence rate among the Scheduled Tribes
- Researchers were working to develop a gene therapy using CRISPR-Cas9
- This comes months after the U.S. Food and Drug Administration approved the CRISPR-Cas9 technology for a cell-based gene therapy to treat sickle cell disease in December 2023.
- Ministry officials said one of the main challenges for India was to find a way to make this therapy cost-effective.
- Developing a gene therapy using CRISPR has been part of India’s mission to eradicate sickle cell disease by 2047.
- Part of this mission is to also conduct over seven crore screenings among vulnerable tribal populations across 17 States and Union Territories, of which three crore screenings have been achieved so far
CRISPR-Cas9
- The CRISPR-Cas9 system consists of an enzyme that behaves like molecular scissors, which can be directed to cut a piece of DNA at a precise location.
- This will then allow a guide RNA to insert a changed genetic code at the sites of the incision.
- While there are a few ways to effect such changes, the CRISPR system is believed to be fast and the most versatile of all.
Prelims takeaway
- CRISPR-Cas9
- Sickle cell disease