AAV5–Factor VIII Gene Transfer in Severe Hemophilia A

Volume: 377, Issue: 26, Pages: 2519 - 2530
Published: Dec 28, 2017
Abstract
Patients with hemophilia A rely on exogenous factor VIII to prevent bleeding in joints, soft tissue, and the central nervous system. Although successful gene transfer has been reported in patients with hemophilia B, the large size of the factor VIII coding region has precluded improved outcomes with gene therapy in patients with hemophilia...
Paper Details
Title
AAV5–Factor VIII Gene Transfer in Severe Hemophilia A
Published Date
Dec 28, 2017
Volume
377
Issue
26
Pages
2519 - 2530
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