First human trial of Gene Therapy in Haemophilia CMC Vellore-India felt proud to conduct the first human clinical trial of gene therapy in Haemophilia A (FVIII deficiency) at Christian Medical College (CMC) Vellore.
Hemophilia is a hemorrhagic disease that includes two distinct genetic disorders caused by missing or defective clotting factor VIII (FVIII; haemophilia A) or clotting factor IX (haemophilia B) source-National Library of Medicine. FVIII is a large protein that is endogenously produced, primarily in the liver by sinusoidal endothelial cells (rather than hepatocytes)
First human trial of Gene Therapy in Haemophilia CMC Vellore
Union Minister of Science & Technology, Dr. Jitendra Singh shared this information here on Wednesday while addressing the “National Science Day 2024” programme in Vigyan Bhavan.
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Department of Biotechnology, Centre for Stem Cell Research – a unit of InStem Bengaluru, in collaboration with Emory University, USA at Christian Medical College, Vellore.is supporting this project Dr Jitendra
This trial involved deploying a novel technology of using a lentiviral vector to express a FVIII transgene in the patient’s haematopoietic stem cell which will then express FVIII from specific differentiated blood cells.