Hemophilia gene therapy: novel rAAV vectors and RNA repair strategy.

  title={Hemophilia gene therapy: novel rAAV vectors and RNA repair strategy.},
  author={Hengjun Chao and C E Walsh},
  journal={Current opinion in molecular therapeutics},
  volume={4 5},
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… CONTINUE READING

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