Therapies for lysosomal storage diseases: Principles, practice, and prospects for refinements based on evolving science.
作者信息
Grabowski Gregory A, Mistry Pramod K
机构信息
University of Cincinnati College of Medicine, Department of Pediatrics, Department of Molecular Genetics, Biochemistry and Microbiology, United States of America; Division of Human Genetics, Cincinnati Children's Hospital Research Foundation, Cincinnati, OH, United States of America.
Yale School of Medicine, Department of Medicine, Department of Pediatrics, Department of Cellular & Molecular Physiology, New Haven, CT, United States of America.
出版信息
Mol Genet Metab. 2022 Sep-Oct;137(1-2):81-91. doi: 10.1016/j.ymgme.2022.07.014. Epub 2022 Jul 30.
PMID:35933791
Abstract
摘要
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