EXTL2 and EXTL3 inhibition with siRNAs as a promising substrate reduction therapy for Sanfilippo C syndrome

Sanfilippo syndrome is a rare lysosomal storage disorder caused by an impaired degradation of heparan sulfate (HS). It presents severe and progressive neurodegeneration and currently there is no effective treatment. Substrate reduction therapy (SRT) may be a useful option for neurological disorders...

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Detalles Bibliográficos
Autores principales: Canals, Isaac, Benetó, Noelia, Cozar, Mónica, Vilageliu, Lluïsa, Grinberg, Daniel
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2015
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4561882/
https://www.ncbi.nlm.nih.gov/pubmed/26347037
http://dx.doi.org/10.1038/srep13654

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4561882/
https://www.ncbi.nlm.nih.gov/pubmed/26347037
http://dx.doi.org/10.1038/srep13654

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