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Synthetic mRNA‐based differentiation method enables early detection of Parkinson's phenotypes in neurons derived from Gaucher disease‐induced pluripotent stem cells
Gaucher disease, the most prevalent metabolic storage disorder, is caused by mutations in the glucocerebrosidase gene GBA1, which lead to the accumulation of glucosylceramide (GlcCer) in affected cells. Gaucher disease type 1 (GD1), although defined as a nonneuronopathic subtype, is accompanied by a...
| Autores principales: | , , , , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
John Wiley & Sons, Inc.
2020
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7980209/ https://www.ncbi.nlm.nih.gov/pubmed/33342090 http://dx.doi.org/10.1002/sctm.20-0302 |