« Previous
Next »
Neuromuscular Disorders
Volume 20, Issue 2
, Pages 102-110
, February 2010
Comparative analysis of antisense oligonucleotide sequences targeting exon 53 of the human DMD gene: Implications for future clinical trials
References
- . Dystrophin: the protein product of the Duchenne muscular dystrophy locus. Cell. 1987;51:919–928
- . An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locus. Genomics. 1988;2:90–95
- . Genetic treatments in muscular dystrophies. Curr Opin Neurol. 2007;20:590–594
- Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse. Proc Natl Acad Sci USA. 2001;98:42–47
- Therapeutic antisense-induced exon skipping in cultured muscle cells from six different DMD patients. Hum Mol Genet. 2003;12:907–914
- Antisense-induced exon skipping restores dystrophin expression in DMD patient derived muscle cells. Hum Mol Genet. 2001;10:1547–1554
- Antisense-induced multiexon skipping for Duchenne muscular dystrophy makes more sense. Am J Hum Genet. 2004;74:83–92
- . Antisense-induced exon skipping for duplications in Duchenne muscular dystrophy. BMC Med Genet. 2007;8:43–51
- Comparative analysis of antisense oligonucleotide sequences for targeted skipping of exon 51 during pre-mRNA splicing in human muscle. Hum Gene Ther. 2007;18:798–810
- Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse. Nat Med. 2003;9:1009–1014
- . Towards a therapeutic inhibition of dystrophin exon 23 splicing in mdx mouse muscle induced by antisense oligonucleotides (splicomers): target sequence optimisation using oligonucleotide arrays. J Gene Med. 2004;6:1149–1158
- Targeted exon skipping in transgenic hDMD mice: a model for direct preclinical screening of human-specific antisense oligonucleotides. Mol Ther. 2004;10:232–240
- Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice. Mol Ther. 2008;16:1624–1629
- Local antisense dystrophin restoration with antisense oligonucleotide PRO051. N Eng J Med. 2007;357:2677–2687
- Restoration of dystrophin expression in Duchenne muscular dystrophy: a single blind, placebo-controlled dose escalation study using morpholino oligomer AVI-4658. Lancet Neurol. 2009;8:918–928
- . Neutrally charged phosphorodiamidate morpholino antisense oligomers: uptake, efficacy and pharmacokinetics. Curr Pharm Biotechnol. 2004;5:431–439
- In vivo comparison of 2′-O-methyl-PS and morpholino antisense oligonucleotides for DMD exon skipping. J Gene Med. 2009;11:257–266
- . Morpholino antisense oligonucleotide induced dystrophin exon 23 skipping in mdx mouse muscle. Hum Mol Genet. 2003;12:1801–1811
- Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology. Nat Med. 2006;12:175–177
- . Dosing regimen has a significant impact on the efficiency of morpholino oligomer-induced exon skipping in mdx mice. Hum Gene Ther. 2009;20:955–965
- Induced dystrophin exon skipping in human muscle explants. Neuromuscul Disord. 2006;16:583–590
- . Antisense oligonucleotide-induced exon skipping restores dystrophin expression in vitro in a canine model of DMD. Gene Ther. 2006;13:1373–1381
- Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations. Hum Mutat. 2009;30:293–299
- . Design of phosphorodiamidate morpholino oligomers (PMOs) for the induction of exon skipping of the human DMD gene. Mol Ther. 2009;17:554–561
- . Antisense oligonucleotide-induced exon skipping across the human dystrophin gene transcript. Mol Ther. 2007;15:1288–1296
- McCulley C, Adkin C, Fletcher S, Wilton SD, Morgan J, Wells DJ. Optimising the efficacy of morpholino antisense oligonucleotide induced exon-skipping in primary myoblasts using nucleofection, submitted for publication.
- Generation and characterization of transgenic mice with the full-length human DMD gene. J Biol Chem. 2008;283:5899–5907
- Functional analysis of 114 exon-internal AONs for targeted DMD exon skipping: indication for steric hindrance of SR protein binding sites. Oligonucleotides. 2005;15:284–297
- Guidelines for antisense oligonucleotide design and insight into splice-modulating mechanisms. Mol Ther. 2008;17:548–553
- . The influence of antisense oligonucleotide length on dystrophin exon skipping. Mol Ther. 2007;15:157–166
- Comparative analysis of antisense oligonucleotide analogs for targeted DMD exon 46 skipping in muscle cells. Gene Ther. 2004;11:1391–1398
PII: S0960-8966(09)00666-X
doi: 10.1016/j.nmd.2009.10.013
© 2009 Elsevier B.V. All rights reserved.
« Previous
Next »
Neuromuscular Disorders
Volume 20, Issue 2
, Pages 102-110
, February 2010
