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Phase I study of liver depot gene therapy in late-onset Pompe disease

2023; Elsevier BV; Volume: 31; Issue: 7 Linguagem: Inglês

10.1016/j.ymthe.2023.02.014

1525-0024

Edward C. Smith, Sam Hopkins, Laura E. Case, Ming Xu, Crista Walters, Stephanie DeArmey, Sang-oh Han, Tracy Spears, Jessica A. Chichester, Edward H. Bossen, Christoph P. Hornik, Jennifer L. Cohen, Deeksha Bali, Priya S. Kishnani, Dwight D. Koeberl,

Adenosine and Purinergic Signaling

Gene therapy with an adeno-associated virus serotype 8 (AAV8) vector (AAV8-LSPhGAA) could eliminate the need for enzyme replacement therapy (ERT) by creating a liver depot for acid α-glucosidase (GAA) production. We report initial safety and bioactivity of the first dose (1.6 × 10