Article

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

2023-02-07

Abstract excerpt

Large genes including several CRISPR-Cas modules, such as 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 r...

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Literature Corpus work: 5db22da2-cafd-571d-8ca5-7c5079feb493
DOI: 10.1101/2023.02.07.527504

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