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Showing 1–2 of 2 results
Advanced filters: Author: Nina Karguth Clear advanced filters

  • Large genes require dual adeno-associated viral (AAV) vectors for in vivo delivery/expression, but current methods have limitations. Here the authors develop and functionally evaluate REVeRT, an efficient and flexible dual AAV vector technology based on reconstitution via mRNA trans-splicing.

    • Lisa Maria Riedmayr
    • Klara Sonnie Hinrichsmeyer
    • Elvir Becirovic
    ResearchOpen Access
    Nature Communications
    Volume: 14, P: 1-14

  • The authors provide a versatile gene therapy approach that is mutation- and gene size–independent, using dCas9-VPR–based transcriptional activation of functionally equivalent genes. They show how to apply this for gene therapy for inherited retinal dystrophies in mice as an example.

    • Lisa M. Riedmayr
    • Klara S. Hinrichsmeyer
    • Elvir Becirovic
    Protocols
    Nature Protocols
    Volume: 17, P: 781-818