Article
Trans-splicing improvement by the combined application of antisense strategies.
International journal of molecular sciences - 6 Jan 2015
Koller Ulrich, Hainzl Stefan, Kocher Thomas, Hüttner Clemens, Klausegger Alfred, Gruber Christina, Mayr Elisabeth, Wally Verena, Bauer Johann W, Murauer Eva M
Abstract excerpt
Spliceosome-mediated RNA trans-splicing has become an emergent tool for the repair of mutated pre-mRNAs in the treatment of genetic diseases. RNA trans-splicing molecules (RTMs) are designed to induce a specific trans-splicing reaction via a binding domain for a respective target pre-mRNA region....
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.
