HEMOPHILIA : SCIENCE & TECHNOLOGY

HEMOPHILIA : SCIENCE & TECHNOLOGY

NEWS: Indian scientists develop novel gene therapy treatment for haemophilia

 

WHAT’S IN THE NEWS?

India's gene therapy trial successfully treated severe hemophilia A by delivering a functional gene using a safe vector, enabling patients to naturally produce clotting Factor VIII, offering a one-time solution. This therapy is safer, potentially suitable for children, and could make treatment affordable in India.

 

 

 

 

Gene Therapy Trial for Hemophilia A in India: Explained

What is Hemophilia A?

  • A genetic disorder where the body is unable to produce Factor VIII, a crucial protein for blood clotting.
  • Leads to frequent and severe bleeding episodes, sometimes life-threatening.
  • Traditional treatment involves frequent injections of synthetic or donor-derived Factor VIII.

 

Breakthrough in India

  • A gene therapy trial conducted in Tamil Nadu successfully treated five patients with severe hemophilia A.
  • The patients have remained free of bleeding episodes for over a year.
  • This therapy could revolutionize hemophilia treatment by offering a one-time solution.

 

Key Features of the Gene Therapy

1. Target

  • Objective: To address the root genetic cause of hemophilia A.
  • Mechanism: Provides a functional copy of the faulty gene, enabling the body to produce Factor VIII naturally.

2. Delivery Mechanism

  • A harmless, modified virus (vector) is used to transport the therapeutic gene into the patient’s body.
  • The vector does not cause any disease.

3. Gene Functionality

  • Once inside the body, the inserted gene starts instructing the patient’s cells to produce Factor VIII.
  • This restores the natural blood clotting process.

4. One-Time Treatment

  • Unlike current treatments requiring lifelong regular injections of clotting factors, this therapy is designed to work with a single administration.

5. Safer Alternative

  • Uses an advanced, safer vector compared to traditional adenovirus-based vectors.
  • May be more suitable for children, expanding its potential application.

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    Why is This Important for India?

    • Cost Reduction: Current hemophilia treatments are extremely expensive and unaffordable for many patients in India.
    • Better Access: A one-time, effective therapy could drastically improve access to care.

    • Enhanced Quality of Life: Reduces the burden of frequent injections and hospital visits for hemophilia patients.