A high-throughput screening to identify small molecules that suppress huntingtin promoter activity or activate huntingtin-antisense promoter activity

Huntington’s disease (HD) is a neurodegenerative disorder caused by a CAG repeat expansion in exon 1 of huntingtin (HTT). While there are currently no disease-modifying treatments for HD, recent efforts have focused on the development of nucleotide-based therapeutics to lower HTT expression. As an a...

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Udgivet i:Sci Rep
Main Authors: Khaled, Houda G., Feng, Hongxuan, Hu, Xin, Sun, Xin, Zheng, Wang, Li, Pan P., Rudnicki, Dobrila D., Ye, Wenjuan, Chen, Yu-Chi, Southall, Noel, Marugan, Juan, Ross, Christopher A., Ferrer, Marc, Henderson, Mark J., Margolis, Russell L.
Format: Artigo
Sprog:Inglês
Udgivet: Nature Publishing Group UK 2021
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7969751/
https://ncbi.nlm.nih.gov/pubmed/33731741
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41598-021-85279-2
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