Ontology highlight
ABSTRACT:
SUBMITTER: Trochet D
PROVIDER: S-EPMC5801507 | biostudies-other | 2018 Feb
REPOSITORIES: biostudies-other
EMBO molecular medicine 20180201 2
Rapid advances in allele-specific silencing by RNA interference established a strategy of choice to cure dominant inherited diseases by targeting mutant alleles. We used this strategy for autosomal-dominant centronuclear myopathy (CNM), a rare neuromuscular disorder without available treatment due to heterozygous mutations in the <i>DNM2</i> gene encoding Dynamin 2. Allele-specific siRNA sequences were developed in order to specifically knock down the human and murine <i>DNM2</i>-mRNA harbouring ...[more]