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.
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.
