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Titel:

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

Dokumenttyp:
Journal Article; Research Support, Non-U.S. Gov't
Autor(en):
Truong, Dong-Jiunn Jeffery; Kühner, Karin; Kühn, Ralf; Werfel, Stanislas; Engelhardt, Stefan; Wurst, Wolfgang; Ortiz, Oskar
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
Volltext / DOI:
doi:10.1093/nar/gkv601
PubMed:
http://view.ncbi.nlm.nih.gov/pubmed/26082496
Print-ISSN:
0305-1048
TUM Einrichtung:
Institut für Pharmakologie und Toxikologie
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