Department of Biotechnology (DBT), along with the Centre for Stem Cell Research (CSCR) develops 1st human gene therapy for Haemophilia A | Current Affairs | Vision IAS
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This Gene therapy used a lentiviral vector to introduce a normal copy of the Factor VIII gene into autologous hematopoietic stem cells (HSCs) to treat Haemophilia A (refer to box). 

  • Lentiviral vectors are a type of viral vector that can be used to transfer genetic material.
  • HSCs are multipotent primitive cells that can develop into all types of blood cells.
Image representing Gene Therapy process

About Gene therapy 

  • Gene therapy is a technique that uses a gene(s) to treat, prevent or cure a disease or medical disorder.
    • It works by replacing faulty genesdeactivating harmful ones, or introducing new genes to improve or restore health.
    • It uses products/methods such as Plasmid DNA (Genetically engineered Circular DNA molecules), Human gene editing technology, etc. 
  • Types of Gene Therapy
  • Germline gene therapy:  In it, the Germline Cell (egg or sperm) are modified by the introduction of functional genes.
  • Somatic cell gene therapy: In this, therapeutic gene are transferred to a patient’s somatic cells (cells other than germline cells). 
  • Application: both inherited genetic diseases (e.g., sickle cell disease) and acquired disorders (e.g., leukemia) could be treated.

About Haemophilia

  • It is a rare condition that affects the blood's ability to clot.
  • India bears the world’s second-largest burden of haemophilia. 
  • Haemophilia A is caused by the deficiency of clotting Factor VIII.
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