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

Document Type

Article

Author

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

Source

In Molecular Therapy 5 July 2023 31(7):1994-2004

Subject

Language

ISSN

1525-0016

Online Access

Full Text (ScienceDirect 종량제) Web of Science JCR 저널정보 Scopus Find it@PNU

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