Somatic gene editing ameliorates skeletal and cardiac muscle failure in pig and human models of Duchenne muscular dystrophy.

Moretti, A

Somatic gene editing ameliorates skeletal and cardiac muscle failure in pig and human models of Duchenne muscular dystrophy. [electronic resource] - Nature medicine 02 2020 - 207-214 p. digital

Publication Type: Journal Article; Research Support, Non-U.S. Gov't

1546-170X

10.1038/s41591-019-0738-2 doi


Animals
Disease Models, Animal
Dystrophin--genetics
Exons
Female
Frameshift Mutation
Gene Editing--methods
Gene Expression Regulation
Genetic Therapy
Genome
Heart Failure--genetics
Humans
Induced Pluripotent Stem Cells--cytology
Male
Mass Spectrometry
Muscle, Skeletal--metabolism
Muscles--metabolism
Muscular Dystrophy, Duchenne--genetics
Myoblasts--metabolism
Myocytes, Cardiac--metabolism
Proteome
Swine
RNA, Guide, CRISPR-Cas Systems