CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells-Reference-Cited by-同舟云学术

CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells

Published:2019-03 Issue:3 Volume:5 Page:
ISSN:2375-2548
Container-title:Science Advances
language:en
Short-container-title:Sci. Adv.

Author:

Min Yi-Li¹²^ORCID,Li Hui¹²^ORCID,Rodriguez-Caycedo Cristina¹²^ORCID,Mireault Alex A.¹²^ORCID,Huang Jian³,Shelton John M.³^ORCID,McAnally John R.¹²^ORCID,Amoasii Leonela¹²⁴,Mammen Pradeep P. A.²³^ORCID,Bassel-Duby Rhonda¹²^ORCID,Olson Eric N.¹²^ORCID

Affiliation:

1. Department of Molecular Biology, Hamon Center for Regenerative Science and Medicine, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA.

2. Sen. Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA.

3. Department of Internal Medicine, University of Texas Southwestern Medical Center, 5323 Harry Hines Boulevard, Dallas, TX 75390, USA.

4. Exonics Therapeutics, 490 Arsenal Way, Watertown, MA 02472, USA.

Abstract

A mouse model with a common human DMD mutation was created and corrected by CRISPR-Cas9 gene editing.

Funder

National Institutes of Health

Welch Foundation

Parent Project Muscular Dystrophy Award

CureDuchenne

Publisher

American Association for the Advancement of Science (AAAS)

Subject

Multidisciplinary

Reference39 articles.

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2. The structural and functional diversity of dystrophin;Ahn A. H.;Nat. Genet.,1993

3. Association of dystrophin and an integral membrane glycoprotein

4. The TREAT-NMD DMD Global Database: Analysis of More than 7,000 Duchenne Muscular Dystrophy Mutations

5. Exon-skipping advances for Duchenne muscular dystrophy;Echevarría L.;Hum. Mol. Genet.,2018

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