In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy-Reference-Cited by-同舟云学术

In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy

Published:2016-01-22 Issue:6271 Volume:351 Page:403-407
ISSN:0036-8075
Container-title:Science
language:en
Short-container-title:Science

Author:

Nelson Christopher E.¹²,Hakim Chady H.³,Ousterout David G.¹²,Thakore Pratiksha I.¹²,Moreb Eirik A.¹²,Rivera Ruth M. Castellanos⁴,Madhavan Sarina¹²,Pan Xiufang³,Ran F. Ann⁵⁶,Yan Winston X.⁵⁷⁸,Asokan Aravind⁴,Zhang Feng⁵⁹¹⁰¹¹,Duan Dongsheng³¹²,Gersbach Charles A.¹²¹³

Affiliation:

1. Department of Biomedical Engineering, Duke University, Durham, NC, USA.

2. Center for Genomic and Computational Biology, Duke University, Durham, NC, USA.

3. Department of Molecular Microbiology and Immunology, University of Missouri, Columbia, MO, USA.

4. Gene Therapy Center, Departments of Genetics, Biochemistry and Biophysics, School of Medicine, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.

5. Broad Institute of MIT and Harvard, Cambridge, MA, USA.

6. Society of Fellows, Harvard University, Cambridge, MA, USA.

7. Graduate Program in Biophysics, Harvard Medical School, Boston, MA, USA.

8. Harvard–MIT Division of Health Sciences and Technology, Harvard Medical School, Boston, MA, USA.

9. McGovern Institute for Brain Research, Massachusetts Institute of Technology, Cambridge, MA, USA.

10. Department of Brain and Cognitive Sciences, Massachusetts Institute of Technology, Cambridge, MA, USA.

11. Department of Biological Engineering, Massachusetts Institute of Technology, Cambridge, MA, USA.

12. Department of Neurology, University of Missouri, Columbia, MO, USA.

13. Department of Orthopaedic Surgery, Duke University Medical Center, Durham, NC, USA.

Abstract

Editing can help build stronger muscles Much of the controversy surrounding the gene-editing technology called CRISPR/Cas9 centers on the ethics of germline editing of human embryos to correct disease-causing mutations. For certain disorders such as muscular dystrophy, it may be possible to achieve therapeutic benefit by editing the faulty gene in somatic cells. In proof-of-concept studies, Long et al. , Nelson et al. , and Tabebordbar et al. used adeno-associated virus-9 to deliver the CRISPR/Cas9 gene-editing system to young mice with a mutation in the gene coding for dystrophin, a muscle protein deficient in patients with Duchenne muscular dystrophy. Gene editing partially restored dystrophin protein expression in skeletal and cardiac muscle and improved skeletal muscle function. Science , this issue p. 400 , p. 403 , p. 407

Funder

Muscular Dystrophy Association

Duke-Coulter Translational Partnership Grant

March of Dimes Foundation

NSF

Keck

Damon Runyon

Searle Scholars

Merkin Family

Vallee

Simons

Paul G. Allen