Selective suppression of polyglutamine-expanded protein by lipid nanoparticle-delivered siRNA targeting CAG expansions in the mouse CNS

Polyglutamine (polyQ) diseases are inherited neurodegenerative disorders caused by expansion of cytosine-adenine-guanine (CAG)-trinucleotide repeats in causative genes. These diseases include spinal and bulbar muscular atrophy (SBMA), Huntington’s disease, dentatorubral-pallidoluysian atrophy, and s...

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Detalhes bibliográficos
Publicado no:Mol Ther Nucleic Acids
Main Authors: Hirunagi, Tomoki, Sahashi, Kentaro, Tachikawa, Kiyoshi, Leu, Angel I., Nguyen, Michelle, Mukthavaram, Rajesh, Karmali, Priya P., Chivukula, Padmanabh, Tohnai, Genki, Iida, Madoka, Onodera, Kazunari, Ohyama, Manabu, Okada, Yohei, Okano, Hideyuki, Katsuno, Masahisa
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2021
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7937577/
https://ncbi.nlm.nih.gov/pubmed/33738134
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2021.02.007
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