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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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  • E-project-042617-181354
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  • 2017
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  • 2017-04-26
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