Treatment of a Mouse Model of ALS by In Vivo Base Editing. [electronic resource]
Producer: 20210406Description: 1177-1189 p. digitalISSN:- 1525-0024
- Amyotrophic Lateral Sclerosis -- genetics
- Animals
- CRISPR-Associated Protein 9 -- metabolism
- Codon, Nonsense
- Dependovirus -- genetics
- Disease Models, Animal
- Gene Editing
- Genetic Vectors -- administration & dosage
- HEK293 Cells
- Humans
- Injections, Spinal
- Inteins
- Male
- Mice
- Mice, Transgenic
- Streptococcus pyogenes -- enzymology
- Superoxide Dismutase-1 -- genetics
- Trans-Splicing
- Treatment Outcome
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Publication Type: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't; Research Support, U.S. Gov't, Non-P.H.S.
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