Successful Phenotype Improvement following Gene Therapy for Severe Hemophilia A in Privately Owned Dogs.

by Mary Beth Callan, Mark E Haskins, Ping Wang, Shangzhen Zhou, Katherine A High, Valder R Arruda
Published 2016-01-01

CRISPR Activation Enhances In Vitro Potency of AAV Vectors Driven by Tissue-Specific Promoters

by Devin S. McDougald, Thu T. Duong, Katherine C. Palozola, Anson Marsh, Tyler E. Papp, Jason A. Mills, Shangzhen Zhou, Jean Bennett
Published 2019-06-01

Bioengineered coagulation factor VIII enables long-term correction of murine hemophilia A following liver-directed adeno-associated viral vector delivery

by Harrison C Brown, J Fraser Wright, Shangzhen Zhou, Allison M Lytle, Jordan E Shields, H Trent Spencer, Christopher B Doering
Published 2014-01-01

Safety of Same-Eye Subretinal Sequential Readministration of AAV2-hRPE65v2 in Non-human Primates

by Lindsey Weed, Michael J. Ammar, Shangzhen Zhou, Zhangyong Wei, Leona W. Serrano, Junwei Sun, Vivian Lee, Albert M. Maguire, Jean Bennett, Tomas S. Aleman
Published 2019-12-01

Comparative AAV-eGFP Transgene Expression Using Vector Serotypes 1–9, 7m8, and 8b in Human Pluripotent Stem Cells, RPEs, and Human and Rat Cortical Neurons

by Thu T. Duong, James Lim, Vidyullatha Vasireddy, Tyler Papp, Hung Nguyen, Lanfranco Leo, Jieyan Pan, Shangzhen Zhou, H. Isaac Chen, Jean Bennett, Jason A. Mills
Published 2019-01-01

Correction: AAV-Mediated Gene Therapy for Choroideremia: Preclinical Studies in Personalized Models.

by Vidyullatha Vasireddy, Jason A Mills, Rajashekhar Gaddameedi, Etiena Basner-Tschakarjan, Monika Kohnke, Aaron D Black, Krill Alexandrov, Shangzhen Zhou, Albert M Maguire, Daniel C Chung, Helen Mac, Lisa Sullivan, Paul Gadue, Jeannette L Bennicelli, Deborah L French, Jean Bennett
Published 2015-01-01

AAV-mediated gene therapy for choroideremia: preclinical studies in personalized models.

by Vidyullatha Vasireddy, Jason A Mills, Rajashekhar Gaddameedi, Etiena Basner-Tschakarjan, Monika Kohnke, Aaron D Black, Krill Alexandrov, Shangzhen Zhou, Albert M Maguire, Daniel C Chung, Helen Mac, Lisa Sullivan, Paul Gadue, Jeannette L Bennicelli, Deborah L French, Jean Bennett
Published 2013-01-01