In a groundbreaking achievement, the Centre for Stem Cell Research (CSCR) at the Christian Medical College, Vellore, in collaboration with the Department of Biotechnology (DBT), has successfully executed India's first human gene therapy for patients with Hemophilia A.
Hemophilia A, a genetic disorder, affects approximately 1 in 10,000 individuals worldwide. It is characterized by the deficiency of factor VIII, a crucial protein necessary for blood clotting. Current treatments involve regular infusions of recombinant factor VIII, which can be expensive and require lifelong administration.
These modified HSCs generate blood cells capable of producing functional Factor VIII over extended periods, thus obviating the need for repeated infusions.
The success of this gene therapy treatment for Hemophilia A patients has reported that it will to improve the lives of thousands of individuals affected by this debilitating disorder, for the development of gene therapies for other genetic disorders.
The novel gene therapy treatment, developed by Indian scientists, involves introducing a functional copy of the factor VIII gene into the patient's liver cells using a viral vector. Scientists opinion enables the liver to produce the deficient protein, thereby alleviating the symptoms of Hemophilia A.
The therapy successfully produced zero annualized bleeding rates in all five enrolled participants while enabling prolonged production of Factor VIII, eliminating the need for repeated infusionsNew England Journal of Medicine
The CSCR and DBT collaboration has been instrumental in developing this innovative treatment. The research team, led by Dr. George Thomas, Director of the CSCR, has worked tirelessly to design and optimize the gene therapy protocol. The DBT has provided significant funding and support for the project, enabling the researchers to translate their findings from the laboratory to the clinic.
Reference:
https://pmc.ncbi.nlm.nih.gov/articles
https://health.economictimes.com/Indias-first-human-gene-therapy
(Input From Various Sources)
(Rehash/Neha Kamble)