Therapeutic reversal of Huntington’s disease by in vivo self-assembled siRNAs

Huntington’s disease is an autosomal-dominant neurodegenerative disease caused by CAG expansion in exon 1 of the huntingtin (HTT) gene. Since mutant huntingtin (mHTT) protein is the root cause of Huntington’s disease, oligonucleotide-based therapeutic approaches using small interfering RNAs (siRNAs)...

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Detalles Bibliográficos
Autores principales: Zhang, Li, Wu, Tengteng, Shan, Yangyang, Li, Ge, Ni, Xue, Chen, Xiaorui, Hu, Xiuting, Lin, Lishan, Li, Yongchao, Guan, Yalun, Gao, Jinfeng, Chen, Dingbang, Zhang, Yu, Pei, Zhong, Chen, Xi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2021
Materias:
Original Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8677541/
https://www.ncbi.nlm.nih.gov/pubmed/34918046
http://dx.doi.org/10.1093/brain/awab354

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