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
Monahan, Paul E., Sun, Junjiang, Gui, Tong, Hu, Genlin, Hannah, William B., Wichlan, David G., Wu, Zhijian, Grieger, Joshua C., Li, Chengwen, Suwanmanee, Thipparat, Stafford, Darrel W., Booth, CarmenVolume:
26
Language:
english
Journal:
Human Gene Therapy
DOI:
10.1089/hum.2014.106
Date:
February, 2015
File:
PDF, 893 KB
english, 2015