title: Current status of gene therapy for hemophilia.
abstract: The hemophilias are an attractive model for gene therapy because their clinical manifestations are attributable to the lack of a single protein that circulates in minute amounts in the plasma. Sustained therapeutic expression of factors VIII and IX has been achieved in preclinical studies using a wide range of gene transfer technologies targeted at different tissues. This achievement has led to six different phase I/II clinical trials that resulted in limited efficacy but minimal toxicity. Recombinant adeno-associated viral vectors appear most promising for hemophilia gene therapy; however, this review summarizes all the major gene therapy approaches used and outlines the future challenges.
authors.affiliation: Department of Haematology, University College London, 98 Chenies Mews, London WCIE 6HX, UK. a.nathwani@ucl.ac.uk
authors.forename: Amit C
authors.initials: AC
authors.lastname: Nathwani
authors.forename: Arthur W
authors.initials: AW
authors.lastname: Nienhuis
authors.forename: Andrew M
authors.initials: AM
authors.lastname: Davidoff
mesh_terms: D005164:Factor IX / Q000235:genetics; D005169:Factor VIII / Q000235:genetics; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors; D006467:Hemophilia A / Q000628:therapy*; D006801:Humans
chemical_list: D005169:Factor VIII; D005164:Factor IX