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

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Posted 29 Feb 2024

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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.

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.