Gene therapy for Haemopholia

About Haemophilia

• It is a rare genetic blood disorder that prevents blood from clotting properly. It’s usually inherited and more common in males.
• Most common types are hemophilia A (classic hemophilia) and hemophilia B (Christmas disease)
• Haemophilia A: It occurs when the body doesn’t produce enough factor VIII, a protein that helps blood clot.
• India has the second-largest patient pool globally, with an estimated 40,000 to 100,000 patients.
• Treatment costs are very high: ₹2.54 crore per patient over 10 years.

Gene Therapy

• It is a medical technique that treats or prevents disease by altering a person’s genes. It can involve:
  • Replacing a disease-causing gene with a healthy copy.
  • Inactivating a disease-causing gene.
  • Introducing a new or modified gene into the body.
  • Training the body’s immune system to recognize and attack cancer cells.
• Gene therapy can be used to treat a variety of diseases, including: Cancer, Genetic diseases like hemophilia and sickle cell disease etc.

Gene Therapy as a One-Time Solution for Haemophilia

• Mechanism: A gene is introduced into the body, enabling it to produce enough Factor VIII to prevent haemorrhage.
• It uses lentivirus as a vector to integrate the clotting factor gene into stem cells, which is later reintroduced into the patient’s body.

Advantages

• Eliminates the need for frequent infusions of clotting factors or other medications.
• Potentially safer for children and avoids the need for immunosuppressive therapy.

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