Gene therapy for haemophilia.

Area of Science:

• Genetics
• Hematology
• Molecular Biology

Background:

• Hemophilia is a genetic bleeding disorder requiring expensive, lifelong clotting factor replacement.
• Gene therapy offers a potential curative treatment for hemophilia.
• This review is an update of a previous Cochrane Review on gene therapy for hemophilia.

Purpose of the Study:

• To evaluate the safety and efficacy of gene therapy for hemophilia A or B.

Main Methods:

• Searched multiple databases and reference lists for relevant clinical trials up to April 17, 2020.
• Included randomized, quasi-randomized, and controlled clinical trials comparing gene therapy to standard treatment.
• Focused on individuals with hemophilia A or B without inhibitors.

Main Results:

• No eligible trials of gene therapy for hemophilia were identified.
• The safety and efficacy of gene therapy for hemophilia could not be determined.

Conclusions:

• No randomized or quasi-randomized clinical trials met the inclusion criteria.
• Well-designed clinical trials are necessary to assess the long-term feasibility, success, and risks of gene therapy for hemophilia.