Targeting Heparan Sulfate Proteoglycans as a Novel Therapeutic Strategy for Mucopolysaccharidoses

Mucopolysaccharidoses (MPSs) are inherited metabolic diseases caused by the deficiency of lysosomal enzymes needed to catabolize glycosaminoglycans (GAGs). Four therapeutic options are currently considered: enzyme replacement therapy, substrate reduction therapy, gene therapy, and hematopoietic stem...

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Bibliografiske detaljer
Udgivet i:Mol Ther Methods Clin Dev
Main Authors: De Pasquale, Valeria, Sarogni, Patrizia, Pistorio, Valeria, Cerulo, Giuliana, Paladino, Simona, Pavone, Luigi Michele
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Gene & Cell Therapy 2018
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6011039/
https://ncbi.nlm.nih.gov/pubmed/29942826
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2018.05.002
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