Enhanced CRISPR-Cas9 correction of Duchenne muscular dystrophy in mice by a self-complementary AAV delivery system-Reference-Cited by-同舟云学术

Enhanced CRISPR-Cas9 correction of Duchenne muscular dystrophy in mice by a self-complementary AAV delivery system

Published:2020-02-21 Issue:8 Volume:6 Page:
ISSN:2375-2548
Container-title:Science Advances
language:en
Short-container-title:Sci. Adv.

Author:

Zhang Yu¹²^ORCID,Li Hui¹²^ORCID,Min Yi-Li¹²^ORCID,Sanchez-Ortiz Efrain¹²^ORCID,Huang Jian³,Mireault Alex A.¹²^ORCID,Shelton John M.³^ORCID,Kim Jiwoong⁴^ORCID,Mammen Pradeep P. A.²³^ORCID,Bassel-Duby Rhonda¹²^ORCID,Olson Eric N.¹²^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. Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.

4. Department of Population and Data Sciences, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.

Abstract

Self-complementary AAV-packaged CRISPR-Cas9 genome editing components rescue Duchenne muscular dystrophy.

Funder

National Institutes of Health

Welch Foundation

Publisher

American Association for the Advancement of Science (AAAS)

Subject

Multidisciplinary

Reference58 articles.

1. Dystrophin: The protein product of the duchenne muscular dystrophy locus

2. Complete cloning of the duchenne muscular dystrophy (DMD) cDNA and preliminary genomic organization of the DMD gene in normal and affected individuals

3. The Dystrophin Complex: Structure, Function, and Implications for Therapy

4. The Pathogenesis and Therapy of Muscular Dystrophies

5. Association of dystrophin and an integral membrane glycoprotein

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