Gene therapy for haemophilia.

Area of Science:

• Hematology
• Genetics
• Medical Research

Background:

• Haemophilia is a genetic bleeding disorder requiring expensive, lifelong clotting factor treatments.
• Gene therapy is being explored as a potential curative treatment for haemophilia A and B.

Purpose of the Study:

• To assess the safety and efficacy of gene therapy for individuals with haemophilia A or B.

Main Methods:

• A systematic search was conducted for randomized or quasi-randomized clinical trials on gene therapy for haemophilia.
• Searches included major databases and reference lists of relevant literature up to November 2014.

Main Results:

• No clinical trials evaluating gene therapy for haemophilia were identified in the search.

Conclusions:

• The current lack of clinical trials prevents an assessment of gene therapy's effects in haemophilia.
• Further well-designed clinical trials are necessary to determine the long-term feasibility, success, and risks of gene therapy for haemophilia.