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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Detalles Bibliográficos
Autores principales: Gaj, Thomas, Ojala, David S., Ekman, Freja K., Byrne, Leah C., Limsirichai, Prajit, Schaffer, David V.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Association for the Advancement of Science 2017
Materias:
Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5738228/
https://www.ncbi.nlm.nih.gov/pubmed/29279867
http://dx.doi.org/10.1126/sciadv.aar3952

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