Toward an Oligonucleotide Therapy for Duchenne Muscular Dystrophy: A Complex Development Challenge-Reference-Cited by-同舟云学术

Toward an Oligonucleotide Therapy for Duchenne Muscular Dystrophy: A Complex Development Challenge

Published:2010-03-31 Issue:25 Volume:2 Page:
ISSN:1946-6234
Container-title:Science Translational Medicine
language:en
Short-container-title:Sci. Transl. Med.

Author:

Wood Matthew J. A.¹

Affiliation:

1. Department of Physiology, Anatomy and Genetics, University of Oxford, Oxford OX1 3QX, UK.

Abstract

By correcting the reading frame in mutant DMD genes, antisense oligonucleotides can restore the production of missing dystrophin protein, but several difficulties must be overcome before such drugs are broadly useful.

Publisher

American Association for the Advancement of Science (AAAS)

Subject

General Medicine

Reference38 articles.

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4. Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study

5. Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse

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