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Article

Generation of New Isogenic Models of Huntington's Disease Using CRISPR-Cas9 Technology.

International journal of molecular sciences - 8 Mar 2020

Dabrowska Magdalena, Ciolak Agata, Kozlowska Emilia, Fiszer Agnieszka, Olejniczak Marta

Abstract excerpt

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 techniques such as genome engineering has generated new opportunities in the field of disease modeling and enabled the...

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Source

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Identifiers

PMID: 32182692
DOI: 10.3390/ijms21051854
PMCID: PMC7084361

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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