Enhanced Enzyme Uptake and Glycogen Clearance in Muscles and CNS of Pompe Mice Administered AAV9 Expressing Novel Engineered GAA
Waijiao Cai, Nick Marze, Erin Lopez, Lucy S. Marie, Ilya Tikh, Susan A. Benard, Vincent Chihyi Yu,Michael St. Andre,Jane M. Owens,Loujin Song, Laura Monarski,Shambhunath Choudhary, Sagar Nisraiyya, Bin Li,Arpana Khatri, Marla Bazile, O. H. Crystal, Kruti Patel,Jatin Narula,Ramesh C. Kovi, John Jakubczak,Lisa R. Keyes, H. Seng Chen,Clark Pan, Sury Somanathan MOLECULAR THERAPY（2023）
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