Targeted Exon Skipping in Transgenic hDMD Mice: A Model for Direct Preclinical Screening of Human-Specific Antisense Oligonucleotides
Open Access
- 31 August 2004
- journal article
- Published by Elsevier BV in Molecular Therapy
- Vol. 10 (2), 232-240
- https://doi.org/10.1016/j.ymthe.2004.05.031
Abstract
No abstract availableKeywords
This publication has 30 references indexed in Scilit:
- Antisense-Induced Multiexon Skipping for Duchenne Muscular Dystrophy Makes More SenseAmerican Journal of Human Genetics, 2004
- Therapeutic antisense-induced exon skipping in cultured muscle cells from six different DMD patientsHuman Molecular Genetics, 2003
- Targeted exon skipping as a potential gene correction therapy for Duchenne muscular dystrophyNeuromuscular Disorders, 2002
- Chimeric snRNA molecules carrying antisense sequences against the splice junctions of exon 51 of the dystrophin pre-mRNA induce exon skipping and restoration of a dystrophin synthesis in Δ48-50 DMD cellsProceedings of the National Academy of Sciences of the United States of America, 2002
- Giant dystrophin deletion associated with congenital cataract and mild muscular dystrophyNeurology, 1998
- Modification of splicing in the dystrophin gene in cultured Mdx muscle cells by antisense oligoribonucleotidesHuman Molecular Genetics, 1998
- Very mild muscular dystrophy associated with the deletion of 46% of dystrophinNature, 1990
- Characterization of Dystrophin in Muscle-Biopsy Specimens from Patients with Duchenne's or Becker's Muscular DystrophyThe New England Journal of Medicine, 1988
- An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locusGenomics, 1988
- Dystrophin: The protein product of the duchenne muscular dystrophy locusCell, 1987