Development of an intein-mediated split–Cas9 system for gene therapy

Using CRISPR/Cas9, it is possible to target virtually any gene in any organism. A major limitation to its application in gene therapy is the size of Cas9 (>4 kb), impeding its efficient delivery via recombinant adeno-associated virus (rAAV). Therefore, we developed a split–Cas9 system, bypassing...

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Bibliographic Details
Published in:Nucleic Acids Res
Main Authors: Truong, Dong-Jiunn Jeffery, Kühner, Karin, Kühn, Ralf, Werfel, Stanislas, Engelhardt, Stefan, Wurst, Wolfgang, Ortiz, Oskar
Format: Artigo
Language:Inglês
Published: Oxford University Press 2015
Subjects:
Molecular Biology
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC4513872/
https://ncbi.nlm.nih.gov/pubmed/26082496
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/nar/gkv601
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