India has conducted the first human clinical trial of gene therapy for haemophilia A (FVIII deficiency) at Christian Medical College (CMC) Vellore.
- The programme is supported by the Department of Biotechnology, the Centre for Stem Cell Research – a unit of InStem Bengaluru.
- The trials involved deploying a novel technology of using a lentiviral vector to express a FVIII transgene in the patient’s own haematopoietic stem cell which will then express FVIII from specific differentiated blood cells.
About Hemophilia and Hemophilia A
- Hemophilia is usually an inherited bleeding disorder in which the blood does not clot properly.
- People with hemophilia have low levels of either factor VIII (8) or factor IX (9).
- Hemophilia A, also called factor VIII (8) deficiency or classic hemophilia, is a genetic disorder caused by missing or defective factor VIII (FVIII), a clotting protein.
- Although it is passed down from parents to children, about 1/3 of cases found have no previous family history.
- Females can also have hemophilia, but this is much rarer.
