Therapies for lysosomal storage diseases: Principles, practice, and prospects for refinements based on evolving science

Mol Genet Metab. 2022 Sep-Oct;137(1-2):81-91. doi: 10.1016/j.ymgme.2022.07.014. Epub 2022 Jul 30.

¹ 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. Electronic address: grabgo317@comcast.net.
² Yale School of Medicine, Department of Medicine, Department of Pediatrics, Department of Cellular & Molecular Physiology, New Haven, CT, United States of America.

No abstract available

Keywords: Glycosphingolipidoses; Lyso-lipids; Mucopolysaccharidoses; Pompe disease; mTORC1.

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