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Document type:
journal article 
Author(s):
Truong, Dong-Jiunn Jeffery; Kühner, Karin; Kühn, Ralf; Werfel, Stanislas; Engelhardt, Stefan; Wurst, Wolfgang; Ortiz, Oskar 
Title:
Development of an intein-mediated split-Cas9 system for gene therapy. 
Abstract:
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 the packaging limit using split-inteins. Each Cas9 half was fused to the corresponding split-intein moiety and, only upon co-expression, the intein-mediated trans-splicing occurs and the full Cas9 protei...    »
 
Journal title abbreviation:
Nucleic Acids Res 
Year:
2015 
Journal volume:
43 
Journal issue:
13 
Pages contribution:
6450-8 
Language:
eng 
Print-ISSN:
0305-1048 
TUM Institution:
Institut für Pharmakologie und Toxikologie