Cardiac Myoediting Attenuates Cardiac Abnormalities in Human and Mouse Models of Duchenne Muscular Dystrophy

Ayhan Atmanli; Andreas C Chai; Miao Cui; Zhaoning Wang; Takahiko Nishiyama; Rhonda Bassel-Duby; Eric N Olson

doi:10.1161/CIRCRESAHA.121.319579

Cardiac Myoediting Attenuates Cardiac Abnormalities in Human and Mouse Models of Duchenne Muscular Dystrophy

Circ Res. 2021 Sep 3;129(6):602-616. doi: 10.1161/CIRCRESAHA.121.319579. Epub 2021 Aug 10.

Authors

Ayhan Atmanli^{1

2

3}, Andreas C Chai^{1

2

3}, Miao Cui^{1

2

3}, Zhaoning Wang^{1

2

3}, Takahiko Nishiyama^{1

2

3}, Rhonda Bassel-Duby^{1

2

3}, Eric N Olson^{1

2

3}

Affiliations

¹ Department of Molecular Biology (A.A., A.C.C., M.C., Z.W., T.N., R.B.-D., E.N.O.), University of Texas Southwestern Medical Center, Dallas, TX.
² Hamon Center for Regenerative Science and Medicine (A.A., A.C.C., M.C., Z.W., T.N., R.B.-D., E.N.O.), University of Texas Southwestern Medical Center, Dallas, TX.
³ Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center (A.A., A.C.C., M.C., Z.W., T.N., R.B.-D., E.N.O.), University of Texas Southwestern Medical Center, Dallas, TX.

Abstract

[Figure: see text].

Publication types

Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't

MeSH terms

Animals
CRISPR-Cas Systems
Cardiomyopathies / etiology
Cardiomyopathies / therapy*
Cell Line
Cells, Cultured
Dependovirus / genetics
Dystrophin / genetics
Dystrophin / metabolism
Gene Editing / methods*
Genetic Therapy / methods*
Humans
Induced Pluripotent Stem Cells / cytology
Induced Pluripotent Stem Cells / metabolism
Male
Mice
Mice, Inbred C57BL
Muscular Dystrophy, Duchenne / complications
Muscular Dystrophy, Duchenne / therapy*
Myocytes, Cardiac / cytology
Myocytes, Cardiac / metabolism
Transcriptome

Substances

Dmd protein, mouse
Dystrophin

Abstract

Publication types

MeSH terms

Substances

Grants and funding