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    Preclinical pharmacology and safety studies to support an AAV9 NGLY1 gene therapy clinical trial for the treatment of NGLY1 deficiency

    Preclinical pharmacology and safety studies to support an AAV9 NGLY1 gene therapy clinical trial for the treatment of NGLY1 deficiency by Lei Zhu, Jennifer W. Cook, Alicia Newton, Selina S. Dwight, Brendan Beahm, Matt Wilsey, William F. Mueller, Becky Schweighardt

    Published 2025-09-01
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