Faculty Advisor

Adams, David S.

Center

University of Massachusetts Medical Center

Abstract

Pompe disease, a storage disease caused by a lack of lysosomal acid alpha-glucosidase (GAA), results in systemic lysosomal glycogen accumulation and respiratory and muscle destruction. We propose that viral gene therapy can lower glycogen accumulation and relieve symptoms in a GAA-/- mouse model. Two viruses were engineered to contain plasmids encoding GAA (AAVB1-GAA or AAV9-GAA), and were injected IV into 90-day old GAA-/- or WT mice. Preliminary data with respiratory, behavioral, and histological assays suggest that both vectors improve the survival and pathology.

Publisher

Worcester Polytechnic Institute

Date Accepted

April 2017

Major

Biology and Biotechnology

Project Type

Major Qualifying Project

Accessibility

Unrestricted

Advisor Department

Biology and Biotechnology

Project Center

University of Massachusetts Medical Center

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