Correction of diverse muscular dystrophy mutations in human engineered heart muscle by single-site genome editing-Reference-Cited by-同舟云学术

Correction of diverse muscular dystrophy mutations in human engineered heart muscle by single-site genome editing

Published:2018-01-05 Issue:1 Volume:4 Page:
ISSN:2375-2548
Container-title:Science Advances
language:en
Short-container-title:Sci. Adv.

Author:

Long Chengzu¹²³⁴^ORCID,Li Hui¹²³^ORCID,Tiburcy Malte⁵⁶,Rodriguez-Caycedo Cristina¹²³^ORCID,Kyrychenko Viktoriia¹²³,Zhou Huanyu¹²³^ORCID,Zhang Yu¹²³^ORCID,Min Yi-Li¹²³^ORCID,Shelton John M.⁷^ORCID,Mammen Pradeep P. A.²³⁷^ORCID,Liaw Norman Y.⁵^ORCID,Zimmermann Wolfram-Hubertus⁵⁶^ORCID,Bassel-Duby Rhonda¹²³^ORCID,Schneider Jay W.²³⁷,Olson Eric N.¹²³^ORCID

Affiliation:

1. Department of Molecular Biology, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.

2. Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.

3. Hamon Center for Regenerative Science and Medicine, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.

4. Leon H. Charney Division of Cardiology, New York University School of Medicine, New York, NY 10016, USA.

5. Institute of Pharmacology and Toxicology, University Medical Center, Georg-August-University Göttingen, Göttingen, 37075, Germany.

6. DZHK (German Center for Cardiovascular Research), partner site Göttingen, Göttingen, Germany.

7. Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.

Abstract

CRISPR-mediated genome editing efficiently corrected cardiac abnormalities associated with DMD in human engineered heart tissue.

Funder

National Institutes of Health

Welch Foundation

Parent Project Muscular Dystrophy

German Research Foundation

Robert A. Welch Foundation

Foundation Leducq

German Center for Cardiovascular Research

Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center

German Federal Ministry for Science and Education

Publisher

American Association for the Advancement of Science (AAAS)

Subject

Multidisciplinary

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