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

• Main Authors: Anna Kotowska-Zimmer (Author), Lukasz Przybyl (Author), Marianna Pewinska (Author), Joanna Suszynska-Zajczyk (Author), Dorota Wronka (Author), Maciej Figiel (Author), Marta Olejniczak (Author)
• Format: Book
• Published: Elsevier, 2022-06-01T00:00:00Z.
• Subjects: article
• Online Access: 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...