Offering hope to around 136,000 Haemophilia A patients in India, the Department of Biotechnology (DBT), along with the Centre for Stem Cell Research (CSCR) at Christian Medical College (CMC), Vellore, announced that they have successfully executed a first-in-human gene therapy using lentiviral vectors for the disease. Haemophilia is a severe bleeding disorder caused by the deficiency of clotting Factor VIII, leading to spontaneous internal and external bleeding episodes. Although rare, India bears the world’s second-largest burden of haemophilia. Current treatments require frequent Factor VIII replacement therapy, which faces challenges such as high costs, intravenous (IV) access in children, and low patient acceptance.The single-centre study, which enrolled five participants aged between 22 and 41 years, showed transformational results, according to scientists involved in the research. “The therapy successfully produced zero annualised bleeding rates in all five enrolled participan...
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