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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 <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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Main Authors: | , , , , , , , , |
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Formato: | Artigo |
Idioma: | Inglês |
Publicado em: |
MDPI AG
2020-11-01
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Colecção: | Journal of Personalized Medicine |
Assuntos: | |
Acesso em linha: | https://www.mdpi.com/2075-4426/10/4/215 |
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