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A Human Induced Pluripotent Stem Cell-Derived Isogenic Model of Huntington’s Disease Based on Neuronal Cells Has Several Relevant Phenotypic Abnormalities
Huntington’s disease (HD) is a severe neurodegenerative disorder caused by a CAG triplet expansion in the first exon of the HTT gene. Here we report the introduction of an HD mutation into the genome of healthy human embryonic fibroblasts through CRISPR/Cas9-mediated homologous recombination. We ver...
Autores principales: | , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2020
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7712151/ https://www.ncbi.nlm.nih.gov/pubmed/33182269 http://dx.doi.org/10.3390/jpm10040215 |