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

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

Abstract 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...

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Autores principales: Houda G. Khaled, Hongxuan Feng, Xin Hu, Xin Sun, Wang Zheng, Pan P. Li, Dobrila D. Rudnicki, Wenjuan Ye, Yu-Chi Chen, Noel Southall, Juan Marugan, Christopher A. Ross, Marc Ferrer, Mark J. Henderson, Russell L. Margolis
Formato: article
Lenguaje:EN
Publicado: Nature Portfolio 2021
Materias:
Medicine
R
Science
Q
Acceso en línea:https://doaj.org/article/e1dbdd8b7e1f49f1934d1a2643d9a73c
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