BIRSA 101: India's 1st Indigenous CRISPR-Based Gene Therapy for Sickle Cell Disease
- TPP
- Nov 21
- 2 min read
CSIR-IGIB’s CRISPR Gene Therapy Breakthrough for Sickle Cell Disease in India.
India has launched its first indigenous CRISPR-based gene therapy for Sickle Cell Disease (SCD).
The therapy is named BIRSA 101.
The therapy is dedicated to Bhagwan Birsa Munda, a renowned tribal freedom fighter.
BIRSA 101 has been developed by the CSIR–Institute of Genomics and Integrative Biology (IGIB).
About Gene Therapy
Gene therapy is a medical technique that uses genes to treat, prevent, or cure diseases.
Gene therapy works by adding new copies of a broken gene in patient cells.
Gene therapy also works by replacing a defective or missing gene with a healthy therapeutic gene.
About CRISPR Technology
CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats.
CRISPR is a genome-editing tool, which means it allows precise modification of DNA sequences.
CRISPR uses a Guide RNA to locate specific DNA segments in the genome.
Guide RNA is designed to bind only to the target genetic sequence.
CRISPR uses the Cas9 protein as a molecular cutting tool.
Cas9 acts like molecular scissors that cut both strands of DNA at the desired location.
About Sickle Cell Disease (SCD)
Sickle Cell Disease is a genetic disorder, meaning it is inherited through genes.
SCD affects haemoglobin, the protein in red blood cells that carries oxygen.
The disease causes red blood cells to become rigid and sickle-shaped.
Sickle-shaped cells lead to blockages in blood flow inside blood vessels.
SCD has a high prevalence among India’s tribal population.
The incidence of SCD is 1 in 86 births among Scheduled Tribes (STs) in India.
Related News: enFnCas9 Platform
An agreement has been signed between CSIR-IGIB and Serum Institute of India Pvt. Ltd..
The agreement aims to develop scalable and affordable therapies for genetic disorders.
These therapies will be based on the enFnCas9 genome-editing platform.
enFnCas9 refers to the engineered Francisella novicida Cas9 (FnCas9) protein.
enFnCas9 is a high-fidelity CRISPR-Cas9 system, which means it has higher accuracy and fewer off-target effects.
The enFnCas9 platform has been developed by CSIR-IGIB.
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