One transgene, two myopathies: an MTM1 ‘cross gene therapy’ for BIN1 deficiency?-Reference-Cited by-同舟云学术

One transgene, two myopathies: an MTM1 ‘cross gene therapy’ for BIN1 deficiency?

Published:2023-09-20 Issue:10 Volume:146 Page:3966-3968
ISSN:0006-8950
Container-title:Brain
language:en
Short-container-title:

Author:

Asgarian Zeinab¹,Siam Ala’a¹,Counsell John R¹^ORCID

Affiliation:

1. Research Department of Targeted Intervention, UCL Division of Surgery and Interventional Science, Charles Bell House , London , UK

Abstract

This scientific commentary refers to ‘MTM1 overexpression prevents and reverts BIN1-related centronuclear myopathy’ by Giraud et al. (https://doi.org/10.1093/brain/awad251).

Funder

GOSH-LifeArc Translational Accelerator Award

Million Dollar Bike Ride Grant

Publisher

Oxford University Press (OUP)

Subject

Neurology (clinical)

Link

https://academic.oup.com/brain/advance-article-pdf/doi/10.1093/brain/awad310/51712761/awad310.pdf

Reference9 articles.

1. MTM1 overexpression prevents and reverts BIN1-related centronuclear myopathy;Giraud;Brain,2023

2. Common pathogenic mechanisms in centronuclear and myotubular myopathies and latest treatment advances;Gómez-Oca;Int J Mol Sci,2021

3. Reducing dynamin 2 expression rescues X-linked centronuclear myopathy;Cowling;J Clin Invest,2014

4. Mice with muscle-specific deletion of Bin1 recapitulate centronuclear myopathy and acute downregulation of dynamin 2 improves their phenotypes;Silva-Rojas;Mol Ther,2022

5. Amphiphysin 2 modulation rescues myotubular myopathy and prevents focal adhesion defects in mice;Lionello;Sci Transl Med,2019

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