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Therapeutic modulation of DMD splicing by blocking exonic splicing enhancer sites with antisense oligonucleotides.
Ann N Y Acad Sci. 2006 Oct; 1082:74-6.AN

Abstract

Antisense oligonucleotides (AONs) can be used to correct the disrupted reading frame of Duchenne muscular dystophy patients (DMD). We have a collection of 121 AONs, of which 79 are effective in inducing the specific skipping of 38 out of the 79 different DMD exons. All AONs are located within exons and were hypothesized to act by steric hindrance of serine-arginine rich (SR) protein binding to exonic splicing enhancer (ESE) sites. Indeed, retrospective in silico analysis of effective versus ineffective AONs revealed that the efficacy of AONs is correlated to the presence of putative ESE sites (as predicted by the ESEfinder and RESCUE-ESE software). ESE predicting software programs are thus valuable tools for the optimization of exon-internal antisense target sequences.

Authors+Show Affiliations

Department of Human Genetics, DMD Genetic Therapy Group, Leiden University Medical Center, 2333 AL Leiden, The Netherlands.No affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info available

Pub Type(s)

Journal Article

Language

eng

PubMed ID

17145928

Citation

Aartsma-Rus, A, et al. "Therapeutic Modulation of DMD Splicing By Blocking Exonic Splicing Enhancer Sites With Antisense Oligonucleotides." Annals of the New York Academy of Sciences, vol. 1082, 2006, pp. 74-6.
Aartsma-Rus A, Janson AA, Heemskerk JA, et al. Therapeutic modulation of DMD splicing by blocking exonic splicing enhancer sites with antisense oligonucleotides. Ann N Y Acad Sci. 2006;1082:74-6.
Aartsma-Rus, A., Janson, A. A., Heemskerk, J. A., De Winter, C. L., Van Ommen, G. J., & Van Deutekom, J. C. (2006). Therapeutic modulation of DMD splicing by blocking exonic splicing enhancer sites with antisense oligonucleotides. Annals of the New York Academy of Sciences, 1082, 74-6.
Aartsma-Rus A, et al. Therapeutic Modulation of DMD Splicing By Blocking Exonic Splicing Enhancer Sites With Antisense Oligonucleotides. Ann N Y Acad Sci. 2006;1082:74-6. PubMed PMID: 17145928.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Therapeutic modulation of DMD splicing by blocking exonic splicing enhancer sites with antisense oligonucleotides. AU - Aartsma-Rus,A, AU - Janson,A A M, AU - Heemskerk,J A, AU - De Winter,C L, AU - Van Ommen,G-J B, AU - Van Deutekom,J C T, PY - 2006/12/6/pubmed PY - 2007/3/6/medline PY - 2006/12/6/entrez SP - 74 EP - 6 JF - Annals of the New York Academy of Sciences JO - Ann N Y Acad Sci VL - 1082 N2 - Antisense oligonucleotides (AONs) can be used to correct the disrupted reading frame of Duchenne muscular dystophy patients (DMD). We have a collection of 121 AONs, of which 79 are effective in inducing the specific skipping of 38 out of the 79 different DMD exons. All AONs are located within exons and were hypothesized to act by steric hindrance of serine-arginine rich (SR) protein binding to exonic splicing enhancer (ESE) sites. Indeed, retrospective in silico analysis of effective versus ineffective AONs revealed that the efficacy of AONs is correlated to the presence of putative ESE sites (as predicted by the ESEfinder and RESCUE-ESE software). ESE predicting software programs are thus valuable tools for the optimization of exon-internal antisense target sequences. SN - 0077-8923 UR - https://www.unboundmedicine.com/medline/citation/17145928/Therapeutic_modulation_of_DMD_splicing_by_blocking_exonic_splicing_enhancer_sites_with_antisense_oligonucleotides_ L2 - https://doi.org/10.1196/annals.1348.058 DB - PRIME DP - Unbound Medicine ER -