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

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...

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Detalles Bibliográficos
Autores principales: Riedmayr, Lisa Maria, Hinrichsmeyer, Klara Sonnie, Thalhammer, Stefan Bernhard, Mittas, David Manuel, Karguth, Nina, Otify, Dina Yehia, Böhm, Sybille, Weber, Valentin Johannes, Bartoschek, Michael David, Splith, Victoria, Brümmer, Manuela, Ferreira, Raphael, Boon, Nanda, Wögenstein, Gabriele Maria, Grimm, Christian, Wijnholds, Jan, Mehlfeld, Verena, Michalakis, Stylianos, Fenske, Stefanie, Biel, Martin, Becirovic, Elvir
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10584818/
https://www.ncbi.nlm.nih.gov/pubmed/37852949
http://dx.doi.org/10.1038/s41467-023-42386-0

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10584818/
https://www.ncbi.nlm.nih.gov/pubmed/37852949
http://dx.doi.org/10.1038/s41467-023-42386-0

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