mRNA trans-splicing dual AAV vectors for (epi)genome editing and gene therapy

mRNA trans-splicing dual AAV vectors for (epi)genome editing and gene therapy

Abstract 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...

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Bibliographic Details
Main Authors: Lisa Maria Riedmayr, Klara Sonnie Hinrichsmeyer, Stefan Bernhard Thalhammer, David Manuel Mittas, Nina Karguth, Dina Yehia Otify, Sybille Böhm, Valentin Johannes Weber, Michael David Bartoschek, Victoria Splith, Manuela Brümmer, Raphael Ferreira, Nanda Boon, Gabriele Maria Wögenstein, Christian Grimm, Jan Wijnholds, Verena Mehlfeld, Stylianos Michalakis, Stefanie Fenske, Martin Biel, Elvir Becirovic
Format: Article
Language:English
Published: Nature Portfolio 2023-10-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-023-42386-0

Internet

https://doi.org/10.1038/s41467-023-42386-0

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