Design of a bioactive small molecule that targets r(AUUCU) repeats in spinocerebellar ataxia 10

Expanded RNA repeats in non-coding region of a gene represent a hallmark of several diseases. Here, the authors identify two small molecules that selectively bind AU repeats and use them to design a compound that targets the pathogenic RNA associated with spinocerebellar ataxia type 10.

Guardado en:
Detalles Bibliográficos
Autores principales: Wang-Yong Yang, Rui Gao, Mark Southern, Partha S. Sarkar, Matthew D. Disney
Formato: article
Lenguaje:EN
Publicado: Nature Portfolio 2016
Materias:
Q
Acceso en línea:https://doaj.org/article/f56a67cc0b8b4f9395ec9cf105810314
Etiquetas: Agregar Etiqueta
Sin Etiquetas, Sea el primero en etiquetar este registro!
Descripción
Sumario:Expanded RNA repeats in non-coding region of a gene represent a hallmark of several diseases. Here, the authors identify two small molecules that selectively bind AU repeats and use them to design a compound that targets the pathogenic RNA associated with spinocerebellar ataxia type 10.