Genetic Substrate Reduction Therapy: A Promising Approach for Lysosomal Storage Disorders

Lysosomal storage diseases are a group of rare genetic disorders characterized by the accumulation of storage molecules in late endosomes/lysosomes. Most of them result from mutations in genes encoding for the catabolic enzymes that ensure intralysosomal digestion. Conventional therapeutic options i...

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Detalles Bibliográficos
Autores principales: Coutinho, Maria Francisca, Santos, Juliana Inês, Matos, Liliana, Alves, Sandra
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2016
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5456330/
https://www.ncbi.nlm.nih.gov/pubmed/28933412
http://dx.doi.org/10.3390/diseases4040033

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5456330/
https://www.ncbi.nlm.nih.gov/pubmed/28933412
http://dx.doi.org/10.3390/diseases4040033

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