Efficacy and long-term safety of CRISPR/Cas9 genome editing in the SOD1-linked mouse models of ALS
Deng et al. assess the effects of CRISPR/Cas9-mediated genome editing in two transgenic mouse models of amyotrophic lateral sclerosis (ALS) for up to 2 years. They find that the genomic editing prevented the development of ALS-like pathology without any notable side-effects, which provides preclinic...
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Autores principales: | , , , , , , , , , , , , , |
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Formato: | article |
Lenguaje: | EN |
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Nature Portfolio
2021
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Materias: | |
Acceso en línea: | https://doaj.org/article/e548fc08a9654fe99fd22baa46408209 |
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