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Article

Single Intramuscular Injection of AAV-shRNA Reduces DNM2 and Prevents Myotubular Myopathy in Mice.

Molecular therapy : the journal of the American Society of Gene Therapy - 4 Apr 2018

Tasfaout Hichem, Lionello Valentina M, Kretz Christine, Koebel Pascale, Messaddeq Nadia, Bitz Deborah, Laporte Jocelyn, Cowling Belinda S

Abstract excerpt

Myotubular myopathy, or X-linked centronuclear myopathy, is a severe muscle disorder representing a significant burden for patients and their families. It is clinically characterized by neonatal and severe muscle weakness and atrophy. Mutations in the myotubularin (MTM1) gene cause myotubular myopathy, and no specific curative treatment is available. We previously found that dynamin 2 (DNM2) is upregulated in...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Source

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Identifiers

PMID: 29506908
DOI: 10.1016/j.ymthe.2018.02.008
PMCID: PMC6080128

Genes

DNM2
MTM1

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 3 Sept 2026.

Related research

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Single Intramuscular Injection of AAV-shRNA Reduces DNM2 and Prevents Myotubular Myopathy in Mice.PMID 29506908Read ↗

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