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In vivo genome editing improves motor function and extends survival in a mouse model of ALS
Amyotrophic lateral sclerosis (ALS) is a fatal and incurable neurodegenerative disease characterized by the progressive loss of motor neurons in the spinal cord and brain. In particular, autosomal dominant mutations in the superoxide dismutase 1 (SOD1) gene are responsible for ~20% of all familial A...
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| Publicado no: | Sci Adv |
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| Main Authors: | , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
American Association for the Advancement of Science
2017
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5738228/ https://ncbi.nlm.nih.gov/pubmed/29279867 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/sciadv.aar3952 |
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