NDLI: In vivo comparison of 2'-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping.

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In vivo comparison of 2'-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping.

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In vivo comparison of 2'-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping.

Content Provider	World Health Organization (WHO)-Global Index Medicus
Author	Heemskerk, Hans A. de Winter, Christa L. de Kimpe, Sjef J. Van Kuik-romeijn, Petra Heuvelmans, Niki Platenburg, Gerard J. Van Ommen, Gert-jan B. Van Deutekom, Judith C. T. Aartsma-Rus, Annemieke
Description	Country affiliation: Netherlands Author Affiliation: Heemskerk HA ( DMD Genetic Therapy Group, Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands.)
Abstract	BACKGROUND: Antisense-mediated exon skipping is a putative treatment for Duchenne muscular dystrophy (DMD). Using antisense oligonucleotides (AONs), the disrupted DMD reading frame is restored, allowing generation of partially functional dystrophin and conversion of a severe Duchenne into a milder Becker muscular dystrophy phenotype. In vivo studies are mainly performed using 2'-O-methyl phosphorothioate (2OMePS) or morpholino (PMO) AONs. These compounds were never directly compared. METHODS: mdx and humanized (h)DMD mice were injected intramuscularly and intravenously with short versus long 2OMePS and PMO for mouse exon 23 and human exons 44, 45, 46 and 51. RESULTS: Intramuscular injection showed that increasing the length of 2OMePS AONs enhanced skipping efficiencies of human exon 45, but decreased efficiency for mouse exon 23. Although PMO induced more mouse exon 23 skipping, PMO and 2OMePS were more comparable for human exons. After intravenous administration, exon skipping and novel protein was shown in the heart with both chemistries. Furthermore, PMO showed lower intramuscular concentrations with higher exon 23 skipping levels compared to 2OMePS, which may be due to sequestration in the extracellular matrix. Finally, two mismatches rendered 2OMePS but not PMO AONs nearly ineffective. CONCLUSIONS: The results obtained in the present study indicate that increasing AON length improves skipping efficiency in some but not all cases. It is feasible to induce exon skipping and dystrophin restoration in the heart after injection of 2OMePS and unconjugated PMO. Furthermore, differences in efficiency between PMO and 2OMePS appear to be sequence and not chemistry dependent. Finally, the results indicate that PMOs may be less sequence specific than 2OMePS.
File Format	HTM / HTML
ISSN	1099498X
Issue Number	3
Volume Number	11
e-ISSN	15212254
Journal	The Journal of Gene Medicine
Language	English
Publisher	Wiley
Publisher Date	2009-03-01
Publisher Place	Great Britain (UK)
Access Restriction	One Nation One Subscription (ONOS)
Subject Keyword	Discipline Molecular Biology Exons Genetics Gene Transfer Techniques Genetic Therapy Methods Muscular Dystrophy, Duchenne Oligonucleotides, Antisense Phosphorothioate Oligonucleotides Animals Base Sequence Humans Mice Mice, Inbred Mdx Molecular Sequence Data Muscle, Skeletal Cytology Metabolism Therapy Myocardium Administration & Dosage Evaluation Studies Journal Article Research Support, Non-u.s. Gov't
Content Type	Text
Resource Type	Article
Subject	Genetics Drug Discovery Molecular Biology Molecular Medicine Genetics (clinical)

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