A CAG repeat-targeting artificial miRNA lowers the mutant huntingtin level in the YAC128 model of Huntington's disease

Among the many proposed therapeutic strategies for Huntington's disease (HD), allele-selective therapies are the most desirable but also the most challenging. RNA interference (RNAi) tools that target CAG repeats selectively reduce the mutant huntingtin level in cellular models of HD. The purpo...

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Detalles Bibliográficos
Autores principales: Kotowska-Zimmer, Anna, Przybyl, Lukasz, Pewinska, Marianna, Suszynska-Zajczyk, Joanna, Wronka, Dorota, Figiel, Maciej, Olejniczak, Marta
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9126840/
https://www.ncbi.nlm.nih.gov/pubmed/35664700
http://dx.doi.org/10.1016/j.omtn.2022.04.031

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9126840/
https://www.ncbi.nlm.nih.gov/pubmed/35664700
http://dx.doi.org/10.1016/j.omtn.2022.04.031

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