title: Gene therapy for haemophilia.
abstract: 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 has recently been prompted as a curative treatment modality. This is an update of a published Cochrane Review.
  
  
  
  To evaluate the safety and efficacy of gene therapy for treating people with haemophilia A or B.
  
  
  
  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 lists of relevant articles and reviews.Date of last search: 18 August 2016.
  
  
  
  Eligible trials include randomised or quasi-randomised clinical trials, including controlled clinical trials comparing gene therapy (with or without standard treatment) with standard treatment (factor replacement) or other 'curative' treatment such as stem cell transplantation for individuals with haemophilia A or B of all ages who do not have inhibitors to factor VIII or IX.
  
  
  
  No trials of gene therapy for haemophilia were found.
  
  
  
  No trials of gene therapy for haemophilia were identified.
  
  
  
  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.
authors.affiliation: St Jude Children's Research Hospital, 262 Danny Thomas Place, MS 260, Memphis, Tennessee, USA, 38105.
authors.forename: Akshay
authors.initials: A
authors.lastname: Sharma
authors.affiliation: South Asian Cochrane Network & Center, Prof. BV Moses Center for Evidence-Informed Health Care and Health Policy, Christian Medical College, Carman Block II Floor, CMC Campus, Bagayam, Vellore, Tamil Nadu, India, 632002.
authors.forename: Manu
authors.initials: M
authors.lastname: Easow Mathew
authors.affiliation: QMed Knowledge Foundation, A-3, Shubham Centre, Cardinal Gracious Road,, Chakala, Andheri East, Mumbai, Maharashtra, India, 400099.
authors.forename: Vasumathi
authors.initials: V
authors.lastname: Sriganesh
authors.affiliation: Hematology Department, St Jude Children's Research Hospital, Hematology MS 800, Room R5006, 262 Danny Thomas Place, Memphis, Tennessee, USA, TN 38105-3678.
authors.forename: Ulrike M
authors.initials: UM
authors.lastname: Reiss
mesh_terms: D015316:Genetic Therapy; D006467:Hemophilia A / Q000628:therapy*; D002836:Hemophilia B / Q000628:therapy*; D006801:Humans