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Induced dystrophin exon skipping in human muscle explants.
Neuromuscul Disord. 2006 Oct; 16(9-10):583-90.ND

Abstract

Antisense oligonucleotide (AO) manipulation of pre-mRNA splicing of the dystrophin gene is showing promise in overcoming Duchenne muscular dystrophy (DMD)-causing mutations. To date, this approach has been limited to studies using animal models or cultured human muscle cells, and evidence that AOs can induce exon skipping in human muscle has yet to be shown. In this study, we used different AO analogues to induce exon skipping in muscle explants derived from normal and DMD human tissue. We propose that inducing exon skipping in human muscle explants is closer to in vivo conditions than cells in monolayer cultures, and may minimize the numbers of participants in Phase I clinical studies to demonstrate proof of principle of exon skipping in human muscle.

Authors+Show Affiliations

Experimental Molecular Medicine Group, Centre for Neuromuscular and Neurological Disorders, University of Western Australia, Nedlands, WA 6009, Australia.No affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info available

Pub Type(s)

Journal Article
Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't

Language

eng

PubMed ID

16919955

Citation

McClorey, G, et al. "Induced Dystrophin Exon Skipping in Human Muscle Explants." Neuromuscular Disorders : NMD, vol. 16, no. 9-10, 2006, pp. 583-90.
McClorey G, Fall AM, Moulton HM, et al. Induced dystrophin exon skipping in human muscle explants. Neuromuscul Disord. 2006;16(9-10):583-90.
McClorey, G., Fall, A. M., Moulton, H. M., Iversen, P. L., Rasko, J. E., Ryan, M., Fletcher, S., & Wilton, S. D. (2006). Induced dystrophin exon skipping in human muscle explants. Neuromuscular Disorders : NMD, 16(9-10), 583-90.
McClorey G, et al. Induced Dystrophin Exon Skipping in Human Muscle Explants. Neuromuscul Disord. 2006;16(9-10):583-90. PubMed PMID: 16919955.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Induced dystrophin exon skipping in human muscle explants. AU - McClorey,G, AU - Fall,A M, AU - Moulton,H M, AU - Iversen,P L, AU - Rasko,J E, AU - Ryan,M, AU - Fletcher,S, AU - Wilton,S D, Y1 - 2006/08/21/ PY - 2006/03/20/received PY - 2006/05/15/revised PY - 2006/05/26/accepted PY - 2006/8/22/pubmed PY - 2006/12/9/medline PY - 2006/8/22/entrez SP - 583 EP - 90 JF - Neuromuscular disorders : NMD JO - Neuromuscul Disord VL - 16 IS - 9-10 N2 - Antisense oligonucleotide (AO) manipulation of pre-mRNA splicing of the dystrophin gene is showing promise in overcoming Duchenne muscular dystrophy (DMD)-causing mutations. To date, this approach has been limited to studies using animal models or cultured human muscle cells, and evidence that AOs can induce exon skipping in human muscle has yet to be shown. In this study, we used different AO analogues to induce exon skipping in muscle explants derived from normal and DMD human tissue. We propose that inducing exon skipping in human muscle explants is closer to in vivo conditions than cells in monolayer cultures, and may minimize the numbers of participants in Phase I clinical studies to demonstrate proof of principle of exon skipping in human muscle. SN - 0960-8966 UR - https://www.unboundmedicine.com/medline/citation/16919955/Induced_dystrophin_exon_skipping_in_human_muscle_explants_ L2 - https://linkinghub.elsevier.com/retrieve/pii/S0960-8966(06)00174-X DB - PRIME DP - Unbound Medicine ER -