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Nature Medicine  9, 997 - 998 (2003)
doi:10.1038/nm0803-997

Skipping to new gene therapies for muscular dystrophy

James G Tidball1 & Melissa J Spencer2

1  James G. Tidball is in the Department of Physiological Science, the Department of Pathology and Laboratory Medicine and the Duchenne Muscular Dystrophy Research Center, David Geffen School of Medicine, University of California, Los Angeles, California 90095, USA. jtidball@physci.ucla.edu

2  Melissa J Spencer is in the Department of Pediatrics and the Duchenne Muscular Dystrophy Research Center, David Geffen School of Medicine, University of California, Los Angeles, California 90095, USA.

An approach that aims to correct RNA improves muscle function in a mouse model of a disease particularly recalcitrant to gene therapy: Duchenne muscular dystrophy (pages 1009−1014).

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REVIEWS
Advances in Duchenne muscular dystrophy gene therapy
Nature Reviews Genetics Review (01 Oct 2003)

RESEARCH
Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse
Nature Medicine Article (01 Aug 2003)

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Nature Medicine
ISSN: 1078-8956
EISSN: 1546-170X
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