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

Author:

Grabowski Gregory A.,Mistry Pramod K.

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology,Biochemistry,Endocrinology, Diabetes and Metabolism

Reference141 articles.

1. The rapidly evolving view of lysosomal storage diseases;Parenti;EMBO Mol. Med.,2021

2. Multi-system disorders of glycosphingolipid and ganglioside metabolism;Xu;J. Lipid Res.,2010

3. Analyses of variant acid beta-glucosidases: effects of Gaucher disease mutations;Liou;J. Biol. Chem.,2006

4. The Online Metabolic and Molecular Bases of Inherited Disease;Grabowski,2006

5. Gaucher disease: basic and translational science needs for more complete therapy and management;Grabowski;Mol. Genet. Metab.,2021

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1. Reduction of lysosome abundance and GAG accumulation after odiparcil treatment in MPS I and MPS VI models;Molecular Genetics and Metabolism Reports;2023-12

2. From Acid Alpha-Glucosidase Deficiency to Autophagy: Understanding the Bases of POMPE Disease;International Journal of Molecular Sciences;2023-08-05

3. Zebra-Sphinx: Modeling Sphingolipidoses in Zebrafish;International Journal of Molecular Sciences;2023-03-01

4. Lysosomal Diseases;Encyclopedia of Cell Biology;2023

5. Targeting the Complement–Sphingolipid System in COVID-19 and Gaucher Diseases: Evidence for a New Treatment Strategy;International Journal of Molecular Sciences;2022-11-18

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