Human Gene Therapy 

Current Context : Recently, Department of Biotechnology (DBT), along with the Centre for Stem Cell Research (CSCR) develops 1st human gene therapy for Haemophilia A

• Uses a lentiviral vector to introduce a normal copy of the Factor VIII gene into autologous hematopoietic stem cells (HSCs).

About Lentiviral Vectors and HSCs:

• Lentiviral Vectors: A type of viral vector used to transfer genetic material into cells.
• Hematopoietic Stem Cells (HSCs): Multipotent cells capable of developing into all blood cell types.

About Gene Therapy :

• Technique using genes to treat, prevent, or cure diseases.
• Methods: Replaces faulty genes, deactivates harmful ones, or introduces new genes.
• Products Used: Plasmid DNA, gene editing technologies (e.g., CRISPR-Cas9).
• Types of Gene Therapy:
  • Germline Gene Therapy: Modifies egg or sperm cells; changes are heritable.
  • Somatic Cell Gene Therapy: Targets non-reproductive cells, affecting only the treated individual.

Significance :

• Treats inherited genetic diseases(e.g., sickle cell disease).
• Addresses acquired disorders(e.g., leukemia).

About Haemophilia:

• Rare disorder affecting blood clotting.
• Prevalence: India bears the second-largest burdenof haemophilia globally.
• Haemophilia A: Caused by a deficiency of clotting Factor VIII.