Gene therapy with adeno-associated virus vector 5–human factor IX in adults with hemophilia B
Abstract
Key Points AAV5 liver-directed wild-type hFIX gene transfer was well tolerated and clinically effective in severe and moderate-severe hemophilia B. No cellular immune responses to the AAV5 vector were detected, and FIX expression levels were stable for the entire observation...
Paper Details
Title
Gene therapy with adeno-associated virus vector 5–human factor IX in adults with hemophilia B
Published Date
Mar 1, 2018
Journal
Volume
131
Issue
9
Pages
1022 - 1031
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Notes
History