Home-grown gene editing tool can make therapy cheaper, effective, Pg13

Key Highlights:

• An indigenously developed gene editing tool from CSIR-IGIB has been transferred to the Serum Institute of India (SII) for genetic disease treatments.
• The therapy, named 'Birsa-101', targets sickle cell disease, prevalent in tribal populations, and is a tribute to Birsa Munda.
• Phase I trials for 'Birsa-101' are expected to begin next year in collaboration with AIIMS, Delhi.
• The indigenous development aims to significantly reduce treatment costs compared to existing therapies like Casgevy, which costs around US $2.2 million.

Detailed Insights:

• The indigenous CRISPR system development began in 2016 at IGIB, with the Ministry of Tribal Affairs recognizing its potential for treating sickle cell disease.
• Sickle cell disease is a genetic condition causing rigid, sickle-shaped red blood cells, leading to pain, organ damage, anemia, infections, and strokes due to blocked blood flow.
• 'Birsa-101' aims to correct the genetic mutations causing sickle cell disease through a one-time infusion, differing from Casgevy which boosts foetal haemoglobin production.
• CRISPR Cas9 gene editing, which won the 2020 Nobel Prize in Chemistry, utilizes bacterial immune system proteins to precisely cut out damaged genetic material.
• A challenge with Cas9 proteins is off-targeting, where unintended genetic material editing can occur, but the new tool aims to minimize this risk.

Scientific/Technical Concepts Involved:

• CRISPR Cas9: A gene editing technology using bacterial proteins to precisely cut and modify DNA sequences.
• Gene editing: A type of genetic engineering in which DNA is inserted, deleted, modified or replaced in the genome of a living organism.
• Sickle cell disease: A genetic blood disorder causing red blood cells to become sickle-shaped, leading to various complications.
• Off-targeting: A phenomenon where gene editing tools modify DNA at unintended locations, potentially causing adverse effects.