Finding a Treatment for ALS - Will Gene Editing Cut It?

N Engl J Med. 2018 Apr 12;378(15):1454-1456. doi: 10.1056/NEJMcibr1716741.

Authors

Ammar Al-Chalabi¹, Robert H Brown Jr¹

Affiliation

¹ From King's College London, Maurice Wohl Clinical Neuroscience Institute, Department of Basic and Clinical Neuroscience, Institute of Psychiatry, Psychology, and Neuroscience, De Crespigny Park, and King's College Hospital, Denmark Hill - both in London (A.A.-C.); and the Department of Neurology, University of Massachusetts Medical School, Worcester (R.H.B.).

PMID: 29641956
DOI: 10.1056/NEJMcibr1716741

No abstract available

MeSH terms

Amyotrophic Lateral Sclerosis / genetics
Amyotrophic Lateral Sclerosis / therapy*
Animals
CRISPR-Cas Systems*
Dependovirus
Disease Models, Animal
Gene Editing*
Gene Expression
Genetic Therapy / methods*
Humans
Mice
Mice, Transgenic
Mutation
Superoxide Dismutase / genetics*

Substances

Superoxide Dismutase

Grants and funding