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...

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Publicat a:J Pers Med
Autors principals: Malankhanova, Tuyana, Suldina, Lyubov, Grigor’eva, Elena, Medvedev, Sergey, Minina, Julia, Morozova, Ksenia, Kiseleva, Elena, Zakian, Suren, Malakhova, Anastasia
Format: Artigo
Idioma:Inglês
Publicat: MDPI 2020
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Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC7712151/
https://ncbi.nlm.nih.gov/pubmed/33182269
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/jpm10040215
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