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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...
Autores principales: | , , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Nature Publishing Group UK
2020
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7244548/ https://www.ncbi.nlm.nih.gov/pubmed/32444599 http://dx.doi.org/10.1038/s41467-020-16318-1 |