Stefanie Fenske

Oct 18, 2023 | nature.com | Lisa Maria Riedmayr |Klara Sonnie Hinrichsmeyer |Michael Bartoschek |Christian Grimm |Jan Wijnholds |Stefanie Fenske | +1 more

AbstractLarge 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).