Truncation of mutant huntingtin in knock-in mice demonstrates exon1 huntingtin is a key pathogenic form

Polyglutamine expansion in proteins can cause selective neurodegeneration, although the mechanisms are not fully understood. In Huntington’s disease (HD), proteolytic processing generates toxic N-terminal huntingtin (HTT) fragments that preferentially kill striatal neurons. Here, using CRISPR/Cas9 t...

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Pubblicato in:Nat Commun
Autori principali: Yang, Huiming, Yang, Su, Jing, Liang, Huang, Luoxiu, Chen, Luxiao, Zhao, Xianxian, Yang, Weili, Pan, Yongcheng, Yin, Peng, Qin, Zhaohui S, Li, Shihua, Li, Xiao-Jiang
Natura: Artigo
Lingua:Inglês
Pubblicazione: Nature Publishing Group UK 2020
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Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC7244548/
https://ncbi.nlm.nih.gov/pubmed/32444599
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41467-020-16318-1
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