학술논문
Antisense oligonucleotide-mediated Dnm2 knockdown prevents and reverts myotubular myopathy in mice
Document Type
article
Author
Source
Nature Communications, Vol 8, Iss 1, Pp 1-13 (2017)
Subject
Language
English
ISSN
2041-1723
Abstract
X-linked myotubular myopathy is caused by mutations in the gene coding for myotubularin 1, and is characterized by overexpression of dynamin 2. Here the authors develop antisense oligonucleotides to dynamin 2, and show that systemic injection leads to improved pathology in mice.