Benutzer: Gast  Login
Dokumenttyp:
journal article 
Autor(en):
Truong, Dong-Jiunn Jeffery; Kühner, Karin; Kühn, Ralf; Werfel, Stanislas; Engelhardt, Stefan; Wurst, Wolfgang; Ortiz, Oskar 
Titel:
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...    »
 
Zeitschriftentitel:
Nucleic Acids Res 
Jahr:
2015 
Band / Volume:
43 
Heft / Issue:
13 
Seitenangaben Beitrag:
6450-8 
Sprache:
eng 
Print-ISSN:
0305-1048 
TUM Einrichtung:
Institut für Pharmakologie und Toxikologie