Session Details

Session Details

01:30 PM - 03:15 PM - Ballroom 4

Steven Gray - PhD - University of Texas Southwestern

Co-Chair

01:30 PM - 03:15 PM - Ballroom 4

Patricia Musolino - Neurology, Massachusetts General Hospital

Co-Chair

01:30 PM - 01:45 PM - Ballroom 4

Xiupeng Chen - pH.D - UMass Chan Medical School

32: Promoter Influences Acute Liver Toxicity and Long-Term Hepatic Genotoxicity in rAAV SMA Gene Therapy in Mice

01:45 PM - 02:00 PM - Ballroom 4

Kelly Glajch - PhD - Biogen

33: Intra-CNS AAV9-GBA1 Delivery Produces Species and Route of Administration Differences in Safety and Efficacy

02:00 PM - 02:15 PM - Ballroom 4

Burcak Ozes - PhD - Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus

34: AAV1.NT-3 Gene Therapy Improves Function, Electrophysiology, and Histopathology in Sh3tc2^−/− Mouse, a CMT4C Model

02:15 PM - 02:30 PM - Ballroom 4

Claire Shamber - Massachusetts General Hospital

35: AAV9-rMOCS1 Gene Therapy, a Bicistronic Gene Delivery, Fully Rescues Neonatally Lethal Mouse Model of Molybdenum Cofactor Deficiency Type A

02:30 PM - 02:45 PM - Ballroom 4

Ruxiao Xing - University of Massachusetts Chan Medical School

36: Developing an AAV-Based Gene Replacement Therapy for Mitochondrial Alanyl-tRNA Synthetase 2 (AARS2) Leukodystrophy

02:45 PM - 03:00 PM - Ballroom 4

Sophia Liu - UMass Chan Medical School

37: Extracellular Vesicles as the Underlying Mechanism for Cross-Correction with rAAV-Based Gene Therapy for GM3 Synthase Deficiency

03:00 PM - 03:15 PM - Ballroom 4

Wu Chen - Baylor College of Medicine

38: AAV Gene Therapy Corrects Neurological Phenotypes with Clinically Relevant Doses in a Mouse Model of STXBP1-Related Developmental and Epileptic Encephalopathy