India conducted the first human clinical trial of gene therapy for hemophilia A (FVIII deficiency): Ministry of Science and Technology | Current Affairs | Vision IAS
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    India conducted the first human clinical trial of gene therapy for hemophilia A (FVIII deficiency): Ministry of Science and Technology

    Posted 29 Feb 2024

    2 min read

    • Trial involved deploying a novel technology of using a lentiviral vector to express a FVIII transgene in the patient’s Hematopoietic stem cell (HSC).
      • HSCs have the capacity to self-renew and the potential to differentiate into all of the mature blood cell types.
    • Trial was conducted by Christian Medical College, Vellore, supported by Department of Biotechnology, along with Emory University, USA etc. 
    • Gene Therapy is a technique that modifies a person’s genes to treat or cure disease.
      • It is a direct way to treat genetic conditions as well as other conditions.
      • Mechanisms
        • Replacing a disease-causing gene with a healthy copy of the gene
        • Inactivating a disease-causing gene that is not functioning properly
        • Introducing a new or modified gene into the body 
      • Key Gene Therapy Products 
        • Plasmid DNA: Circular DNA molecules can be genetically engineered to carry therapeutic genes into human cells.
        • Viral Vectors: Gene therapy products derived from viruses can be used as vectors (vehicles) to carry therapeutic genes.
        • Other: Patient-derived cellular gene therapy products, Bacterial vectors, Human gene editing technology etc. 
      • Key Applications: Treating genetic disorders (sickle cell disease etc.), Cancer treatment (E.g. - CAR T-cell therapy) etc.

    About Hemophilia

    • Hemophilia is usually an inherited bleeding disorder in which the blood does not clot properly. 
    • It is caused by a mutation or change, in one of the genes, that provides instructions for making the clotting factor proteins needed to form a blood clot.
    • This can lead to spontaneous bleeding as well as bleeding following injuries or surgery.  
    • Tags :
    • gene therapy for hemophilia A
    • Gene Therapy
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