Truncation of mutant huntingtin in knock-in mice demonstrates exon1 huntingtin is a key pathogenic form
The mechanisms by which mutant Huntington protein Htt leads to selective neurodegeneration are not fully understood. Here, using gene editing in HD140Q knock-in mice, the authors show that exon1 Htt is a critical pathological form of the protein.
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Auteurs principaux: | , , , , , , , , , , , , |
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Format: | article |
Langue: | EN |
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Nature Portfolio
2020
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Accès en ligne: | https://doaj.org/article/f7ec8fe55f5148dab5116e4f227b70c1 |
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Résumé: | The mechanisms by which mutant Huntington protein Htt leads to selective neurodegeneration are not fully understood. Here, using gene editing in HD140Q knock-in mice, the authors show that exon1 Htt is a critical pathological form of the protein. |
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