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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Detalles Bibliográficos
Autores principales: De Pasquale, Valeria, Sarogni, Patrizia, Pistorio, Valeria, Cerulo, Giuliana, Paladino, Simona, Pavone, Luigi Michele
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2018
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6011039/
https://www.ncbi.nlm.nih.gov/pubmed/29942826
http://dx.doi.org/10.1016/j.omtm.2018.05.002

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6011039/
https://www.ncbi.nlm.nih.gov/pubmed/29942826
http://dx.doi.org/10.1016/j.omtm.2018.05.002

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