Gene Therapy for Pompe Disease Using AAV9 and AAVB1 Encoding the GAA Gene

Bircsak, Samantha Rose

Student Work

Gene Therapy for Pompe Disease Using AAV9 and AAVB1 Encoding the GAA Gene

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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.

This report represents the work of one or more WPI undergraduate students submitted to the faculty as evidence of completion of a degree requirement. WPI routinely publishes these reports on its website without editorial or peer review.

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