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 <i>HTT</i> gene. Here we report the introduction of an HD mutation into the genome of healthy human embryonic fibroblasts through CRISPR/Cas9-mediated homologous re...

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Auteurs principaux: Tuyana Malankhanova, Lyubov Suldina, Elena Grigor’eva, Sergey Medvedev, Julia Minina, Ksenia Morozova, Elena Kiseleva, Suren Zakian, Anastasia Malakhova
Format: Artigo
Langue:Inglês
Publié: MDPI AG 2020-11-01
Collection:Journal of Personalized Medicine
Sujets:
Huntington’s disease modeling
genome editing
induced pluripotent stem cells
Accès en ligne:https://www.mdpi.com/2075-4426/10/4/215
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