Ontology highlight
ABSTRACT:
SUBMITTER: Riedmayr LM
PROVIDER: S-EPMC10584818 | biostudies-literature | 2023 Oct
REPOSITORIES: biostudies-literature
Riedmayr Lisa Maria LM Hinrichsmeyer Klara Sonnie KS Thalhammer Stefan Bernhard SB Mittas David Manuel DM Karguth Nina N Otify Dina Yehia DY Böhm Sybille S Weber Valentin Johannes VJ Bartoschek Michael David MD Splith Victoria V Brümmer Manuela M Ferreira Raphael R Boon Nanda N Wögenstein Gabriele Maria GM Grimm Christian C Wijnholds Jan J Mehlfeld Verena V Michalakis Stylianos S Fenske Stefanie S Biel Martin M Becirovic Elvir E
Nature communications 20231018 1
Large genes including several CRISPR-Cas modules like gene activators (CRISPRa) require dual adeno-associated viral (AAV) vectors for an efficient in vivo delivery and expression. Current dual AAV vector approaches have important limitations, e.g., low reconstitution efficiency, production of alien proteins, or low flexibility in split site selection. Here, we present a dual AAV vector technology based on reconstitution via mRNA trans-splicing (REVeRT). REVeRT is flexible in split site selection ...[more]