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Haemophilia A

Facts for Prelims (FFP)

 

Source: IE

Context: India has successfully conducted the first human clinical trial for gene therapy targeting ‘haemophilia A’.

  • The trials utilized a novel technology involving a lentiviral vector to express an FVIII transgene in the patient’s own haematopoietic stem cell, leading to the expression of FVIII from specific differentiated blood cells.

 

Hemophilia A, also known as classical haemophilia or factor VIII deficiency, is a hereditary bleeding disorder. It’s caused by a mutation on the X-chromosome’s Factor VIII gene, which means that only boys are affected. In most cases, the mutation is passed down from parent to child.