Why in News?
The CSIR-IGIB has developed an indigenous CRISPR-Cas9 gene editing system. It aims to provide low-cost gene therapy for genetic disorders such as sickle cell disease, with prices far lower than existing Western therapies like Casgevy.
Description:
About the Indigenous Gene Editing Tool:
- Developed completely in India by CSIR-Institute of Genomics and Integrative Biology (IGIB).
- Uses the CRISPR-Cas9 mechanism, like the Nobel Prize-winning technology.
- All patents are held by Indian scientists → self-reliance in biotechnology.
Name of the therapy: BIRSA-101:
- Named after Birsa Munda, as sickle cell disease is prevalent in tribal populations.
How CRISPR-Cas9 Works:
- CRISPR = Clustered Regularly Interspaced Short Palindromic Repeats
- Cas9 = endonuclease enzyme acting as molecular scissors
- Uses:
- A guide RNA (gRNA) that binds to a specific DNA sequence.
- The Cas9 protein cuts the DNA at that exact point.
- Once DNA is cut, the cell’s natural repair mechanisms:
- Delete defective sequences,
- Insert corrected ones,
- or Modify problematic genes.
Indigenous vs Western Therapy:
- Western therapy example: Casgevy
- Cost: ~USD 2.2 million per patient
- Targets blood stem cells to produce more fetal haemoglobin (HbF)
- Indian therapy: Expected to cost a small fraction of the above. Designed using indigenous CRISPR system (2016) created by IGIB
- Indian system avoids expensive foreign IP licensing.
Target Disease: Sickle Cell Anaemia:
- Genetic condition causing:
- Crescent-shaped (sickle) RBCs
- Blockage in blood vessels
- Severe pain, anaemia, organ damage, stroke
- India’s large tribal population has high prevalence.
How BIRSA-101 works:
- Corrects the defective gene responsible for sickling.
- Aims to provide a one-time curative therapy.
What is “Off-targeting”?
- When CRISPR edits unintended regions of DNA besides the target sequence.
- Can cause harmful or unpredictable genetic changes.
- Minimizing off-target effects is a major challenge in gene editing.
Advantages of Indigenous CRISPR Platform:
- Low cost
- Scalable manufacturing through Serum Institute
- Reduces India’s dependence on foreign biotech
- Enables gene therapy access for wider population
- Supports Atmanirbhar Bharat in advanced biotechnology.
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