A multifunctional AAV–CRISPR–Cas9 and its host response

Packaging split Cas9 into AAVs increases cargo capacity and allows for efficient genome editing and gene activation in vivo . AAV–split-Cas9 activates the host immune system but does not trigger the extensive cellular damage observed with delivery of Cas9 via DNA electroporation. CRISPR–Cas9 deliver...

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Veröffentlicht in:	Nature methods 2016-10, Vol.13 (10), p.868-874
Hauptverfasser:	Chew, Wei Leong, Tabebordbar, Mohammadsharif, Cheng, Jason K W, Mali, Prashant, Wu, Elizabeth Y, Ng, Alex H M, Zhu, Kexian, Wagers, Amy J, Church, George M
Format:	Artikel
Sprache:	eng
Schlagworte:	631/1647/1511 631/1647/1513/1967 631/250 631/61/338 692/308/2778 Adeno-associated virus Animals Bioengineering Bioinformatics Biological Microscopy Biological Techniques Biomedical Engineering/Biotechnology Caspases CRISPR-Cas Systems - genetics Dependovirus - genetics Dependoviruses Enzyme-Linked Immunosorbent Assay Gene Editing Gene therapy Gene Transfer Techniques Genetic aspects Genetic Engineering - methods Genetic Vectors - genetics HEK293 Cells Humans Immunogenicity Life Sciences Mice Mice, Inbred C57BL Physiological aspects Physiology Proteomics Reverse Transcriptase Polymerase Chain Reaction Viruses
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