Gene therapy for haemophilia.

ABSTRACT:

Background

Haemophilia is a genetic disorder characterized by spontaneous or provoked, often uncontrolled, bleeding into joints, muscles and other soft tissues. Current methods of treatment are expensive, challenging and involve regular administration of clotting factors. Gene therapy for haemophilia is a curative treatment modality currently under investigation. This is an update of a published Cochrane Review.

Objectives

To evaluate the safety and efficacy of gene therapy for treating people with haemophilia A or B.

Search methods

We searched the Cochrane Cystic Fibrosis & Genetic Disorders Group's Coagulopathies Trials Register, compiled from electronic database searches and handsearching of journals and conference abstract books. We also searched the reference lis

SUBMITTER: Sharma A 

PROVIDER: S-EPMC7192367 | biostudies-literature | 2020 Apr

REPOSITORIES: biostudies-literature