Natural History of Infantile GM2 Gangliosidosis

     (PDF, 565 KB)

Dramatic Phenotypic Improvement after Adeno-Associated Virus Gene Therapy in a Feline Model

of Sandhoff Disease

Long-Term Phenotypic Correction of Feline Lysosomal Storage Disease by Intracranial AAV Gene

Therapy

UMASS Medical School Researcher Named Outstanding New Investigator by  The American Society of Gene & Cell Therapy

Effective gene therapy in an authentic model of Tay-Sachs-related diseases

     (PDF, 2.1 MB)

Comparative Analysis of Brain Lipids in Mice, Cats, and Humans with Sandhoff Disease

Rena C. Baek, Douglas R. Martin, Nancy R. Cox, Thomas N. Seyfried

     (PDF, 300 KB)

Published Articles