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Cell‐Based Therapy for Canavan Disease Using Human iPSC‐Derived NPCs and OPCs
Canavan disease (CD) is a fatal leukodystrophy caused by mutation of the aspartoacylase (ASPA) gene, which leads to deficiency in ASPA activity, accumulation of the substrate N‐acetyl‐L‐aspartate (NAA), demyelination, and spongy degeneration of the brain. There is neither a cure nor a standard treat...
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| Publicado no: | Adv Sci (Weinh) |
|---|---|
| Main Authors: | , , , , , , , , , , , , , , , , , , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
John Wiley and Sons Inc.
2020
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7709977/ https://ncbi.nlm.nih.gov/pubmed/33304759 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/advs.202002155 |
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