ASO-Mediated DNM2 Knockdown for Centronuclear Myopathies


Centronuclear myopathies (CNM) are a group of severe muscle diseases for which no effective therapy is currently available. We have previously shown that reduction of the large GTPase DNM2 in a mouse model of the X-linked form, due to loss of myotubularin phosphatase MTM1, prevents the development of the skeletal muscle pathophysiology. As DNM2 is mutated in autosomal dominant forms, here we tested whether DNM2 reduction can rescue DNM2-related CNM in a knock-in mouse harboring the p. R465W mutation (Dnm2RW/+) and displaying a mild CNM phenotype similar to patients with the same mutation.

To download a PDF copy of the presentation, click on the “LinkedIn SlideShare” icon located in the bottom-right corner of the slide-viewer. From the SlideShare landing page click the “Download” button to retrieve the file.

This Virtual Poster has been made available to the scientific community by Aurora Scientific, Inc.

Aurora Scientific, Inc.

Aurora Scientific supports the scientific community in its goal of research and discovery by providing precision instrumentation of the highest quality design, construction and functionality for Muscle Physiology, Material Science and Neuroscience applications.

Aurora Scientific, Inc.

Additional Content From Aurora Scientific, Inc.

Related Content