Open AccessEmploying 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
Author(s) -
Paul E. Monahan,
Junjiang Sun,
Tong Gui,
Genlin Hu,
William B. Hannah,
David Wichlan,
Zhijian Wu,
Joshua C. Grieger,
Chengwen Li,
Thipparat Suwanmanee,
Darrel W. Stafford,
Carmen J. Booth,
Jade Samulski,
Tal Kafri,
Scott McPhee,
R. Jude Samulski
Publication year - 2015
Publication title -
carolina digital repository (university of north carolina at chapel hill)
Language(s) - English
DOI - 10.17615/fzyc-s786
Subject(s) - adeno associated virus , factor ix , genetic enhancement , clinical trial , gain of function , medicine , gene , oncology , bioinformatics , biology , genetics , vector (molecular biology) , mutation , recombinant dna
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