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

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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Bibliographic Details
Main Authors: Anna Kotowska-Zimmer, Lukasz Przybyl, Marianna Pewinska, Joanna Suszynska-Zajczyk, Dorota Wronka, Maciej Figiel, Marta Olejniczak
Format: Article
Language:English
Published: Elsevier 2022-06-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT: Oligonucleotides: Therapies and Applications
RNAi
artificial miRNA
shRNA
CAG repeats
Huntington's disease
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253122001081

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http://www.sciencedirect.com/science/article/pii/S2162253122001081

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