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Small molecule 1a reduces FMRpolyG-mediated toxicity in in vitro and in vivo models for FMR1 premutation

Fragile X-associated tremor and ataxia syndrome (FXTAS) is a late-onset, progressive neurodegenerative disorder characterized by tremors, ataxia and neuropsychological problems. This disease is quite common in the general population with approximately 20 million carriers worldwide. The risk of devel...

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Detalles Bibliográficos
Autores principales: Haify, Saif N, Buijsen, Ronald A M, Verwegen, Lucas, Severijnen, Lies-Anne W F M, de Boer, Helen, Boumeester, Valerie, Monshouwer, Roos, Yang, Wang-Yong, Cameron, Michael D, Willemsen, Rob, Disney, Matthew D, Hukema, Renate K
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8369842/
https://www.ncbi.nlm.nih.gov/pubmed/34077515
http://dx.doi.org/10.1093/hmg/ddab143