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,
Ting Gui,
Gang 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