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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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| Publicado no: | Int J Mol Sci |
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| Main Authors: | , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
MDPI
2019
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6928934/ https://ncbi.nlm.nih.gov/pubmed/31771289 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/ijms20235897 |
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