Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy-Reference-Cited by-同舟云学术

Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy

Published:2017-11-29 Issue:418 Volume:9 Page:
ISSN:1946-6234
Container-title:Science Translational Medicine
language:en
Short-container-title:Sci. Transl. Med.

Author:

Amoasii Leonela¹^ORCID,Long Chengzu¹^ORCID,Li Hui¹^ORCID,Mireault Alex A.¹^ORCID,Shelton John M.²^ORCID,Sanchez-Ortiz Efrain¹^ORCID,McAnally John R.¹,Bhattacharyya Samadrita¹,Schmidt Florian³^ORCID,Grimm Dirk³^ORCID,Hauschka Stephen D.⁴,Bassel-Duby Rhonda¹^ORCID,Olson Eric N.¹^ORCID

Affiliation:

1. Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA.

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

3. Heidelberg University Hospital, Center for Infectious Diseases, Virology, Cluster of Excellence Cell Networks, DZIF partner, BioQuant Center, Heidelberg D-69120, Germany.

4. Department of Biochemistry, University of Washington, Seattle, WA 98195, USA.

Abstract

Single-cut correction of a dystrophin gene mutation with CRISPR/Cas9 restored dystrophin expression in skeletal and cardiac muscles in a mouse model of Duchenne muscular dystrophy.

Funder

NIH Office of the Director

Welch Foundation

Muscular Dystrophy Association

Publisher

American Association for the Advancement of Science (AAAS)

Subject

General Medicine

Reference48 articles.

1. Dystrophin and Muscular Dystrophy: Past, Present, and Future

2. Dystrophin and mutations: one gene, several proteins, multiple phenotypes

3. Muscular dystrophies