Prevention of Dystrophic Pathology in Severely Affected Dystrophin/Utrophin-deficient Mice by Morpholino-oligomer-mediated Exon-skipping-Reference-Cited by-同舟云学术

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Prevention of Dystrophic Pathology in Severely Affected Dystrophin/Utrophin-deficient Mice by Morpholino-oligomer-mediated Exon-skipping

Published:2010-01 Issue:1 Volume:18 Page:198-205
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Goyenvalle Aurélie,Babbs Arran,Powell Dave,Kole Ryszard,Fletcher Sue,Wilton Steve D,Davies Kay E

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference34 articles.

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

2. Exon skipping and translation in patients with frameshift deletions in the dystrophin gene;Sherratt;Am J Hum Genet,1993

3. Modification of splicing in the dystrophin gene in cultured Mdx muscle cells by antisense oligoribonucleotides;Dunckley;Hum Mol Genet,1998

4. Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse;Mann;Proc Natl Acad Sci USA,2001

5. Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse;Lu;Nat Med,2003

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1. Morpholino oligonucleotide-mediated exon skipping for DMD treatment: Past insights, present challenges and future perspectives;Journal of Biosciences;2023-10-11

2. Molecular Genetic Therapies in the Muscular Dystrophies;Current Clinical Neurology;2023

3. Non-uniform dystrophin re-expression after CRISPR-mediated exon excision in the dystrophin/utrophin double-knockout mouse model of DMD;Molecular Therapy - Nucleic Acids;2022-12

4. Non-uniform dystrophin re-expression after CRISPR-mediated exon excision in the dystrophin/utrophin double-knockout mouse model of DMD;2022-01-25

5. Control of backbone chemistry and chirality boost oligonucleotide splice switching activity;Nucleic Acids Research;2022-01-21

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