Details

Autor(en) / Beteiligte

• Liemberger, Bernadette
• Piñón Hofbauer, Josefina
• Wally, Verena
• Arzt, Claudia
• Hainzl, Stefan
• Kocher, Thomas
• Murauer, Eva M
• Bauer, Johann W
• Reichelt, Julia
• Koller, Ulrich

Titel

RNA Trans-Splicing Modulation via Antisense Molecule Interference

Ist Teil von

• International journal of molecular sciences, 2018-03, Vol.19 (3), p.762

Ort / Verlag

Switzerland: MDPI AG

Erscheinungsjahr

2018

Quelle

MEDLINE

Beschreibungen/Notizen

• In recent years, RNA -splicing has emerged as a suitable RNA editing tool for the specific replacement of mutated gene regions at the pre-mRNA level. Although the technology has been successfully applied for the restoration of protein function in various genetic diseases, a higher -splicing efficiency is still desired to facilitate its clinical application. Here, we describe a modified, easily applicable, fluorescence-based screening system for the generation and analysis of antisense molecules specifically capable of improving the RNA reprogramming efficiency of a selected -specific RNA -splicing molecule. Using this screening procedure, we identified several antisense RNAs and short rationally designed oligonucleotides, which are able to increase the -splicing efficiency. Thus, we assume that besides the RNA -splicing molecule, short antisense molecules can act as splicing modulators, thereby increasing the -splicing efficiency to a level that may be sufficient to overcome the effects of certain genetic predispositions, particularly those associated with dominantly inherited diseases.