India makes big breakthrough human gene therapy in treating genetic disorder Hemophilia A

India has achieved a major breakthrough in treating severe Hemophilia A through gene therapy. In a groundbreaking study conducted by the Centre for Stem Cell Research at Christian Medical College, Vellore, five patients who received the therapy experienced zero bleeding episodes over an extended period. The therapy enabled the participants to produce Factor VIII, a protein critical for blood clotting, without needing repeated infusions. UNDERSTANDING HEMOPHILIA A Hemophilia A is a rare genetic disorder caused by a deficiency of blood clotting protein called Factor VIII. In the study, blood stem cells from the patients were modified to include the gene responsible for producing Factor VIII.