Development of an In Vitro Biopotency Assay for an AAV8 Hemophilia B Gene Therapy Vector Suitable for Clinical Product Release

by Johannes Lengler, Sogue Coulibaly, Bernadette Gruber, Reinhard Ilk, Josef Mayrhofer, Friedrich Scheiflinger, Werner Hoellriegl, Falko G. Falkner, Hanspeter Rottensteiner
Published 2020-06-01

Minimal Essential Human Factor VIII Alterations Enhance Secretion and Gene Therapy Efficiency

by Wenjing Cao, Biao Dong, Franziska Horling, Jenni A. Firrman, Johannes Lengler, Matthias Klugmann, Maurus de la Rosa, Wenman Wu, Qizhao Wang, Hongying Wei, Andrea R. Moore, Sean A. Roberts, Carmen J. Booth, Werner Hoellriegl, Dong Li, Barbara Konkle, Carol Miao, Birgit M. Reipert, Friedrich Scheiflinger, Hanspeter Rottensteiner, Weidong Xiao
Published 2020-12-01