An orally available, brain penetrant, small molecule lowers huntingtin levels by enhancing pseudoexon inclusion
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Title
An orally available, brain penetrant, small molecule lowers huntingtin levels by enhancing pseudoexon inclusion
Authors
Keywords
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Journal
Nature Communications
Volume 13, Issue 1, Pages -
Publisher
Springer Science and Business Media LLC
Online
2022-03-03
DOI
10.1038/s41467-022-28653-6
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Note: Only part of the references are listed.- Temporal Phenotypic Changes in Huntington’s Disease Models for Preclinical Studies
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- Silencing Mutant Huntingtin by Adeno-Associated Virus-Mediated RNA Interference Ameliorates Disease Manifestations in the YAC128 Mouse Model of Huntington's Disease
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- Full-Length Human Mutant Huntingtin with a Stable Polyglutamine Repeat Can Elicit Progressive and Selective Neuropathogenesis in BACHD Mice
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