Multiple exon skipping strategies to by-pass dystrophin mutations-Reference-Cited by-同舟云学术

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Multiple exon skipping strategies to by-pass dystrophin mutations

Published:2012-04 Issue:4 Volume:22 Page:297-305
ISSN:0960-8966
Container-title:Neuromuscular Disorders
language:en
Short-container-title:Neuromuscular Disorders

Author:

Adkin Carl F.,Meloni Penelope L.,Fletcher Susan,Adams Abbie M.,Muntoni Francesco,Wong Brenda,Wilton Steve D.

Publisher

Elsevier BV

Subject

Genetics (clinical),Neurology (clinical),Neurology,Pediatrics, Perinatology and Child Health

Reference40 articles.

1. Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology;Alter;Nat Med,2006

2. Morpholino oligomer-mediated exon skipping averts the onset of dystrophic pathology in the mdx mouse;Fletcher;Mol Ther,2007

3. Antisense oligonucleotide-induced exon skipping restores dystrophin expression in vitro in a canine model of DMD;McClorey;Gene Ther,2006

4. Prevention of dystrophic pathology in severely affected dystrophin/utrophin-deficient mice by morpholino-oligomer-mediated exon-skipping;Goyenvalle;Mol Ther,2010

5. Local dystrophin restoration with antisense oligonucleotide PRO051;van Deutekom;N Engl J Med,2007

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4. Developing DMD therapeutics: a review of the effectiveness of small molecules, stop-codon readthrough, dystrophin gene replacement, and exon-skipping therapies;Expert Opinion on Investigational Drugs;2021-01-06

5. The potential of antisense oligonucleotide therapies for inherited childhood lung diseases;Molecular and Cellular Pediatrics;2018-02-06

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