First human trial of Gene Therapy in Haemophilia CMC Vellore


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, 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.

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The Minister showed confidence that manufacturing of this vector will commence soon in India and proceed with further clinical trials. Stating that the National Science Day commemorates the discovery of the “Raman Effect” by Nobel Laureate Sir CV Raman, Dr Jitendra Singh recalled C.V. Raman’s words that India could progress only through science, more science and still more science, and said that after Prime Minister Shri Narendra Modi took over,

On the increasing role of women scientists due to equal opportunity in S&T, Dr Jitendra categorically mentioned that the women scientific community has been empowered under PM Modi as many of the Scientific Institutions and programmes, including Space Missions, are women-led. Dr. Jitendra said that in the wake of recent scientific achievements, it may be underscored that the rising trajectory of India over the past 10 years is being witnessed globally.

“We are globally among the top five countries in scientific research publications, 40th in Global Innovation Index (GII) showcasing remarkable climb from 81st rank in 2015 and our patent filing has crossed 90,000 which is the highest in two decades,” he said.

Dr Jitendra Singh also unveiled a compendium of SATHI Clusters by felicitating heads of IIT Hyderabad, BITS Pilani, ICT Mumbai etc. He also released a White paper on ‘Evolution: Catalysing technology-led ecosystem for Bharat e-Mobility on this occasion

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