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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Detalles Bibliográficos
Autores principales: 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: Online Artículo Texto
Lenguaje:English
Publicado: Springer International Publishing 2020
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7904544/
https://www.ncbi.nlm.nih.gov/pubmed/32696070
http://dx.doi.org/10.1007/s00018-020-03596-7

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