No randomised or quasi-randomised clinical trials of gene therapy for haemophilia were identified. Thus, we are unable to determine the safety and efficacy of gene therapy for haemophilia. Gene therapy for haemophilia is still in its nascent stages and there is a need for well-designed clinical trials to assess the long-term feasibility, success and risks of gene therapy for people with haemophilia.
Wednesday, December 21, 2016
Gene therapy for haemophilia
Cochrane: We reviewed the evidence about the safety and effectiveness of gene therapy for treating people with haemophilia A or haemophilia B. Haemophilia is a bleeding disorder caused by defective genes that produce abnormal blood clotting proteins. Gene therapy modifies or replaces these defective genes with normal ones. We looked for trials that used this approach of modifying or replacing defective genes for producing normal blood clotting proteins for the treatment of haemophilia. This is an update of a published Cochrane Review.We found no trials to provide reliable evidence about the risks or benefits of gene therapy for haemophilia. There is a need for trials that assess the long-term feasibility, success and risks of gene therapy for people with haemophilia.