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Generation of New Isogenic Models of Huntington’s Disease Using CRISPR-Cas9 Technology
Huntington’s disease (HD) is a fatal neurodegenerative disorder caused by the expansion of CAG repeats in exon 1 of the huntingtin gene (HTT). Despite its monogenic nature, HD pathogenesis is still not fully understood, and no effective therapy is available to patients. The development of new techni...
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/PMC7084361/ https://www.ncbi.nlm.nih.gov/pubmed/32182692 http://dx.doi.org/10.3390/ijms21051854 |