Advances in Sphingolipidoses: CRISPR-Cas9 Editing as an Option for Modelling and Therapy

Sphingolipidoses are inherited genetic diseases characterized by the accumulation of glycosphingolipids. Sphingolipidoses (SP), which usually involve the loss of sphingolipid hydrolase function, are of lysosomal origin, and represent an important group of rare diseases among lysosomal storage disord...

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Detalles Bibliográficos
Autores principales: Santos, Renato, Amaral, Olga
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2019
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6928934/
https://www.ncbi.nlm.nih.gov/pubmed/31771289
http://dx.doi.org/10.3390/ijms20235897

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6928934/
https://www.ncbi.nlm.nih.gov/pubmed/31771289
http://dx.doi.org/10.3390/ijms20235897

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