Clinical phenotypes as predictors of the outcome of skipping aroundDMDexon 45-Reference-Cited by-同舟云学术

Clinical phenotypes as predictors of the outcome of skipping aroundDMDexon 45

Published:2015-03-02 Issue:4 Volume:77 Page:668-674
ISSN:0364-5134
Container-title:Annals of Neurology
language:en
Short-container-title:Ann Neurol.

Author:

Findlay Andrew R.¹,Wein Nicolas¹,Kaminoh Yuuki¹,Taylor Laura E.¹,Dunn Diane M.²,Mendell Jerry R.¹³⁴,King Wendy M.⁴,Pestronk Alan⁵,Florence Julaine M.⁵,Mathews Katherine D.⁶,Finkel Richard S.⁷,Swoboda Kathryn J.⁸,Howard Michael T.²,Day John W.⁹,McDonald Craig¹⁰,Nicolas Aurélie¹¹¹²,Le Rumeur Elisabeth¹¹¹²,Weiss Robert B.²,Flanigan Kevin M.¹³⁴,

Affiliation:

1. Center for Gene Therapy, Nationwide Children's Hospital; Columbus OH

2. Department of Human Genetics; University of Utah School of Medicine; Salt Lake City UT

3. Departments of Pediatrics; Ohio State University; Columbus OH

4. Departments of Neurology; Ohio State University; Columbus OH

5. Department of Neurology; Washington University at St Louis; St Louis MO

6. Department of Pediatrics and Neurology; University of Iowa Carver College of Medicine; Iowa City IA

7. Division of Neurology; Nemours Children's Hospital; Orlando FL

8. Department of Neurology; University of Utah School of Medicine; Salt Lake City UT

9. Department of Neurology; University of Minnesota; Minneapolis MN

10. Department of Physical Medicine and Rehabilitation; University of California; Davis Sacramento CA

11. University of Rennes; Rennes France

12. Rennes Institute of Genetics and Development, National Center for Scientific Research; Rennes France

Funder

NIH National Institute of Neurologic Disorders and Stroke

Association Française contre les Myopathies

Publisher

Wiley

Subject

Neurology (clinical),Neurology

Reference31 articles.

1. The muscular dystrophies;Emery;Lancet,2002

2. Improvement of survival in Duchenne Muscular Dystrophy: retrospective analysis of 835 patients;Passamano;Acta Myol,2012

3. An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locus;Monaco;Genomics,1988

4. Advances in Duchenne muscular dystrophy gene therapy;Deutekom;Nat Rev Genet,2003

5. Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study;Cirak;Lancet,2011

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