A non-viral CRISPR/Cas9 delivery system for therapeutically targeting HBV DNA and pcsk9 in vivo

Dear Editor, The clustered regularly interspaced short palindromic repeat （CRISPR）/CRISPR-associated protein （Cas） system has revolutionized biomedical research and facilitated the development of new therapies based on genome editing [1]. A major roadblock to achieve the therapeutic potential of the...

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Veröffentlicht in:	Cell research 2017-03, Vol.27 (3), p.440-443
Hauptverfasser:	Jiang, Chao, Mei, Miao, Li, Bin, Zhu, Xiurui, Zu, Wenhong, Tian, Yujie, Wang, Qiannan, Guo, Yong, Dong, Yizhou, Tan, Xu
Format:	Artikel
Sprache:	eng
Schlagworte:	631/1647/1513/1967 631/1647/1513/1967/3196 631/1647/2300 631/1647/350/354 Animals Base Sequence Biomedical and Life Sciences Cell Biology CRISPR-Associated Protein 9 - metabolism CRISPR-Cas Systems - genetics DNA, Viral - metabolism Gene Targeting HEK293 Cells Hepatitis B virus - genetics Humans Letter to the Editor Life Sciences Lipids - chemistry Liver - metabolism Mice Mice, Inbred C57BL Nanoparticles - chemistry NIH 3T3 Cells Proprotein Convertase 9 - metabolism
Online-Zugang:	Volltext
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Zusammenfassung:	Dear Editor, The clustered regularly interspaced short palindromic repeat （CRISPR）/CRISPR-associated protein （Cas） system has revolutionized biomedical research and facilitated the development of new therapies based on genome editing [1]. A major roadblock to achieve the therapeutic potential of the CRISPR/Cas system is the lack of a safe and effectiving in vivo delivery method.
ISSN:	1001-0602 1748-7838
DOI:	10.1038/cr.2017.16