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Article

In vivo genome editing improves motor function and extends survival in a mouse model of ALS.

Science advances - 1 Dec 2017

Gaj Thomas, Ojala David S, Ekman Freja K, Byrne Leah C, Limsirichai Prajit, Schaffer David V

Abstract excerpt

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 ALS cases. The clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated (Cas9) genome...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Source

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Identifiers

PMID: 29279867
DOI: 10.1126/sciadv.aar3952
PMCID: PMC5738228

Genes

SOD1

Variants

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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