Therapies for lysosomal storage diseases: Principles, practice, and prospects for refinements based on evolving science
Author:
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
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1. Engineering Synthetic and Recombinant Human Lysosomal b-Glucocerebrosidase for Enzyme Replacement Therapy for Gaucher Disease;2024-07-19
2. Reduction of lysosome abundance and GAG accumulation after odiparcil treatment in MPS I and MPS VI models;Molecular Genetics and Metabolism Reports;2023-12
3. From Acid Alpha-Glucosidase Deficiency to Autophagy: Understanding the Bases of POMPE Disease;International Journal of Molecular Sciences;2023-08-05
4. Zebra-Sphinx: Modeling Sphingolipidoses in Zebrafish;International Journal of Molecular Sciences;2023-03-01
5. Lysosomal Diseases;Encyclopedia of Cell Biology;2023
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