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Article

Antisense oligonucleotide-mediated Dnm2 knockdown prevents and reverts myotubular myopathy in mice.

Nature communications - 7 Jun 2017

Tasfaout Hichem, Buono Suzie, Guo Shuling, Kretz Christine, Messaddeq Nadia, Booten Sheri, Greenlee Sarah, Monia Brett P, Cowling Belinda S, Laporte Jocelyn

Abstract excerpt

Centronuclear myopathies (CNM) are non-dystrophic muscle diseases for which no effective therapy is currently available. The most severe form, X-linked CNM, is caused by myotubularin 1 (MTM1) loss-of-function mutations, while the main autosomal dominant form is due to dynamin2 (DNM2) mutations. We previously showed that genetic reduction of DNM2 expression in Mtm1 knockout (Mtm1KO) mice prevents development of...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Muscle Contraction

Share to Muscle, Skeletal

Share to Myopathies, Structural, Congenital

Share to Oligonucleotides, Antisense

Share to Protein Tyrosine Phosphatases, Non-Receptor

Source

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Identifiers

PMID: 28589938
DOI: 10.1038/ncomms15661
PMCID: PMC5467247

Genes

DNM2
MTM1

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026. Manifest publication-graph-cohort-2026-09-06.1.

Source receipt: pubmed, retrieved 6 Sept 2026.

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Antisense oligonucleotide-mediated Dnm2 knockdown prevents and reverts myotubular myopathy in mice.PMID 28589938Read ↗

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