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India is getting closer to developing a gene therapy for sickle cell disease, a genetic blood disorder with a high prevalence rate among the Scheduled Tribes, officials of the Union Tribal Affairs Ministry said
M. Srinivas, Director of the All India Institute of Medical Sciences (AIIMS), said researchers were working to develop a gene therapy using CRISPR-Cas9, a gene-editing tool.
What is CRISPR-Cas9?
CRISPR/Cas9 is a gene-editing technology which involves two essential components: a guide RNA to match a desired target gene, and Cas9 (CRISPR-associated protein 9)—an endonuclease which causes a double-stranded DNA break, allowing modifications to the genome
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.
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