BIRSA 101: India’s 1st Indigenous CRISPR-Based Gene Therapy for Sickle Cell Disease

• 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.