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

• Autori principali: Anna Kotowska-Zimmer (Autore), Lukasz Przybyl (Autore), Marianna Pewinska (Autore), Joanna Suszynska-Zajczyk (Autore), Dorota Wronka (Autore), Maciej Figiel (Autore), Marta Olejniczak (Autore)
• Natura: Libro
• Pubblicazione: Elsevier, 2022-06-01T00:00:00Z.
• Soggetti: article
• Accesso online: Connect to this object online.

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...