Employing a Gain-of-Function Factor IX Variant R338L to Advance the Efficacy and Safety of Hemophilia B Human Gene Therapy: Preclinical Evaluation Supporting an Ongoing Adeno-Associated Virus Clinical Trial

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Title
Employing a Gain-of-Function Factor IX Variant R338L to Advance the Efficacy and Safety of Hemophilia B Human Gene Therapy: Preclinical Evaluation Supporting an Ongoing Adeno-Associated Virus Clinical Trial
Authors
Keywords
-
Journal
HUMAN GENE THERAPY
Volume 26, Issue 2, Pages 69-81
Publisher
Mary Ann Liebert Inc
Online
2014-11-25
DOI
10.1089/hum.2014.106

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