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

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 a...

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Detalhes bibliográficos
Publicado no:Cell Mol Life Sci
Main Authors: 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
Formato: Artigo
Idioma:Inglês
Publicado em: Springer International Publishing 2020
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7904544/
https://ncbi.nlm.nih.gov/pubmed/32696070
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s00018-020-03596-7
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