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Phase 1/2 Study Update of an AAV9-based Gene Therapy for Gaucher Disease Type 2 (PROVIDE Trial)

Sarah Neuhaus, Travis B. Lewis, Paul A. Tamburri,Chester B. Whitley,Deepa Rajan,Renata C. Gallagher,Simon Jones,Aimee Donald,Maria L. Escolar, Yael Beckerman,Erin Mahoney,Daniel A. Hatch, Lee Shaughnessy,Patricia Sondergaard,Olga Uspenskaya,Jeffrey Sevigny

Molecular Genetics and Metabolism（2023）

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