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Article

Artificial miRNAs targeting CAG repeat expansion in ORFs cause rapid deadenylation and translation inhibition of mutant transcripts.

Cellular and molecular life sciences : CMLS - 1 Feb 2021

Ciesiolka Adam, Stroynowska-Czerwinska Anna, Joachimiak Paweł, Ciolak Agata, Kozlowska Emilia, Michalak Michal, Dabrowska Magdalena, Olejniczak Marta, Raczynska Katarzyna D, Zielinska Dominika, Wozna-Wysocka Magdalena, Krzyzosiak Wlodzimierz J, Fiszer Agnieszka

Abstract excerpt

Polyglutamine (polyQ) diseases are incurable neurological disorders caused by CAG repeat expansion in the open reading frames (ORFs) of specific genes. This type of mutation in the HTT gene is responsible for Huntington's disease (HD). CAG repeat-targeting artificial miRNAs (art-miRNAs) were shown as attractive therapeutic approach for polyQ disorders as they caused allele-selective decrease in the level of...

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Identifiers

PMID: 32696070
DOI: 10.1007/s00018-020-03596-7
PMCID: PMC7904544

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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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