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The length of uninterrupted CAG repeats in stem regions of repeat disease associated hairpins determines the amount of short CAG oligonucleotides that are toxic to cells through RNA interference

Extended CAG trinucleotide repeats (TNR) in the genes huntingtin (HTT) and androgen receptor (AR) are the cause of two progressive neurodegenerative disorders: Huntington’s disease (HD) and Spinal and Bulbar Muscular Atrophy (SBMA), respectively. Anyone who inherits the mutant gene in the complete p...

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Detalles Bibliográficos
Autores principales: Murmann, Andrea E., Patel, Monal, Jeong, Si-Yeon, Bartom, Elizabeth T., Jennifer Morton, A., Peter, Marcus E.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9803637/
https://www.ncbi.nlm.nih.gov/pubmed/36585400
http://dx.doi.org/10.1038/s41419-022-05494-1