AAV-based Neonatal Gene Therapy for Hemophilia A: Long-Term Correction and Avoidance of Immune Responses in Mice

por Hu, Chuhong, Lipshutz, Gerald S.
Publicado 2012

Augmentation of Transgene-Encoded Protein After Neonatal Injection of Adeno-Associated Virus Improves Hepatic Copy Number Without Immune Responses

por Tai, Denise S., Hu, Chuhong, Kim, Elizabeth H., Lipshutz, Gerald S.
Publicado 2015

Minimal Ureagenesis is Necessary for Survival in the Murine Model of Hyperargininemia Treated by AAV-based Gene Therapy

por Hu, Chuhong, Tai, Denise S., Park, Hana, Cantero-Nieto, Gloria, Chan, Emily, Yudkoff, Marc, Cederbaum, Stephen D., Lipshutz, Gerald S.
Publicado 2014

AAV-based Gene Therapy Prevents Neuropathology and Results in Normal Cognitive Development in the Hyperargininemic Mouse

por Lee, Eun K., Hu, Chuhong, Bhargava, Ragini, Ponnusamy, Ravi, Park, Hana, Novicoff, Sarah, Rozengurt, Nora, Marescau, Bart, De Deyn, Pater, Stout, David, Schlichting, Lisa, Grody, Wayne W., Cederbaum, Stephen D., Lipshutz, Gerald S.
Publicado 2013