Hemophilia gene therapy: novel rAAV vectors and RNA repair strategy.
Hemophilia results from a deficiency of coagulation Factor VIII or IX and manifests clinically as spontaneous bleeding into the large joints and soft tissue. Current treatment relies on the intravenous infusion of recombinant or purified Factor proteins. Factor infusion is effective, but transient due to the short half-life of Factor proteins. Recent developments in gene transfer technology have led to new strategies using molecular therapeutics as permanent treatment for bleeding disorders. This review describes recent novel molecular strategies for the treatment of the hemophilias.
Chao
Hengjun
H
UNC Gene Therapy Center, Department of Medicine, University of North Carolina, Chapel Hill 27599, USA.
Walsh
Christopher E
CE
D000229:Dependovirus / Q000235:genetics*; D015316:Genetic Therapy; D005822:Genetic Vectors; D006467:Hemophilia A / Q000628:therapy*; D006801:Humans; D012313:RNA / Q000235:genetics*; D012326:RNA Splicing
D012313:RNA