Exploring the Frontiers of Therapeutic Exon Skipping for Duchenne Muscular Dystrophy by Double Targeting within One or Multiple Exons
Open Access
- 30 September 2006
- journal article
- Published by Elsevier in Molecular Therapy
- Vol. 14 (3), 401-407
- https://doi.org/10.1016/j.ymthe.2006.02.022
Abstract
No abstract availableKeywords
This publication has 21 references indexed in Scilit:
- Antisense-Induced Multiexon Skipping for Duchenne Muscular Dystrophy Makes More SenseAmerican Journal of Human Genetics, 2004
- Advances in Duchenne muscular dystrophy gene therapyNature Reviews Genetics, 2003
- Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouseNature Medicine, 2003
- Therapeutic antisense-induced exon skipping in cultured muscle cells from six different DMD patientsHuman Molecular Genetics, 2003
- The muscular dystrophiesThe Lancet, 2002
- Antisense-induced exon skipping restores dystrophin expression in DMD patient derived muscle cellsHuman Molecular Genetics, 2001
- Characterization of Dystrophin in Muscle-Biopsy Specimens from Patients with Duchenne's or Becker's Muscular DystrophyNew 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
- Complete cloning of the duchenne muscular dystrophy (DMD) cDNA and preliminary genomic organization of the DMD gene in normal and affected individualsCell, 1987