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A peptide inhibitor that rescues polyglutamine-induced synaptic defects and cell death through suppressing RNA and protein toxicities

Polyglutamine (polyQ) diseases, including spinocerebellar ataxias and Huntington’s disease, are progressive neurodegenerative disorders caused by CAG triplet-repeat expansion in the coding regions of disease-associated genes. In this study, we found that neurotoxic small CAG (sCAG) RNA species, micr...

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Detalles Bibliográficos
Autores principales: Peng, Shaohong Isaac, Leong, Lok I., Sun, Jacquelyne Ka-Li, Chen, Zhefan Stephen, Chow, Hei-Man, Chan, Ho Yin Edwin
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9240964/
https://www.ncbi.nlm.nih.gov/pubmed/35795484
http://dx.doi.org/10.1016/j.omtn.2022.06.004