India getting close to developing gene therapy for sickle cell disease, say officials

Sub: Science and tech

Sec: Health

Context:

India is getting closer to developing a gene therapy for sickle cell disease which is a genetic blood disorder with a high prevalence rate among the Scheduled Tribes.

More on news:

The National Conclave on Generating Awareness on Sickle Cell Disease was organized by the Tribal Affairs Ministry in collaboration with the Birsa Munda Centre at the AIIMS.
Developing a gene therapy using CRISPR has been part of India’s mission to eradicate sickle cell disease by 2047.
A government dossier on the mission, which was launched by Prime Minister Narendra Modi in July 2023.
Part of this mission is to also conduct over seven crore screenings among vulnerable tribal populations across 17 States and Union Territories.

What is Sickle Cell Anemia?

The genetic error in sickle cell disease leads to red blood cells assuming a crescent shape.
Unlike the disc shaped normal cells, the sickle-like cells cannot move around easily in the vessels, resulting in blocked blood flow.
This can lead to episodes of severe pain, life-threatening infections, anemia, or a stroke.
An estimated 30,000-40,000 children in India are born with the disorder every year.
The symptoms manifest in people who inherit a pair of damaged genes from both parents.
Those who carry only one copy of the gene from one parent can lead a normal life.
This is the same as thalassaemia, in which people who inherit a pair of genes from both parents experience symptoms like severe anemia.

About the CRISPR CAS9 technology:

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.

Other efforts taken:

Apart from the gene therapy being developed by India, the sickle cell disease eradication mission also includes developing two coded formulations — AYUSH-RP and AYUSH-SC3 — for managing the disease through a systemic drug development process.
For this continued testing will be undertaken by the Central Council for Research in Ayurvedic Sciences in collaboration with the Indian Council of Medical Research.