Ontology highlight
ABSTRACT:
SUBMITTER: Giacomotto J
PROVIDER: S-EPMC4468906 | biostudies-literature | 2015 Jun
REPOSITORIES: biostudies-literature
Giacomotto Jean J Rinkwitz Silke S Becker Thomas S TS
Nature communications 20150608
Although zebrafish is used to model human diseases through mutational and morpholino-based knockdown approaches, there are currently no robust transgenic knockdown tools. Here we investigate the knockdown efficiency of three synthetic miRNA-expressing backbones and show that these constructs can downregulate a sensor transgene with different degrees of potency. Using this approach, we reproduce spinal muscular atrophy (SMA) in zebrafish by targeting the smn1 gene. We also generate different tran ...[more]