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Search theses and dissertations gathered from participating repositories worldwide. Every result links back to the library that holds it. No account is needed.

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Showing 1 to 3 of 3 for “"pompe disease"”.

  1. 3D Human Skeletal Muscle Model for Studying Satellite Cell Quiescence and Pompe disease

    Wang, Jason2021

    … muscle biology in the areas of regeneration and disease. In muscle regeneration, satellite cells (SCs) are essential for new muscle fiber formation; however, they lose their native quiescent state upon isolation, making in vitro studies of human SC function challenging. To optimally promote SC …

    duke Repository record

  2. Utilization of Lyso-Phosphatidylserine Nanoparticles to Reduce Immunogenicity and Improve Clinical Utility of Acid Alpha-Glucosidase in the Treatment of Pompe Disease

    Dingman, RobertState University of New York at Buffalo2025

    Ph.D.

    buffalo Repository record

  3. Cross-Species Evolution of New AAV Variants

    Gonzalez, Trevor John2023

    … (GAA) transgene in a mouse model of Pompe disease and enhanced restoration of dystrophin through CRISPR/Cas9 gene editing in the mdx mouse model of Duchenne Muscular Dystrophy using AAV.cc47 vectors. We discovered another cross-species compatible variant (AAV.cc84) with enhanced CNS …

    duke Repository record