Inactivation of Hepatitis B Virus Replication in Cultured Cells and In Vivo with Engineered Transcription Activator-Like Effector Nucleases
TLDR
Efficacy in vivo indicates that these engineered nucleases have potential for use in treatment of chronic HBV infection and are the first to demonstrate a targeted nuclease-mediated disruption of HBV cccDNA.About:
This article is published in Molecular Therapy.The article was published on 2013-10-01 and is currently open access. It has received 220 citations till now. The article focuses on the topics: cccDNA & Transcription activator-like effector nuclease.read more
Citations
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A guide to genome engineering with programmable nucleases
Hyongbum Kim,Jin-Soo Kim +1 more
TL;DR: Known nuclease-specific features are essential for researchers to choose the most appropriate tool for a range of applications, including their composition, targetable sites, specificities and mutation signatures, among other characteristics.
Journal ArticleDOI
Therapeutic genome editing: prospects and challenges
TL;DR: In this article, the authors discuss current progress toward developing programmable nuclease-based therapies as well as future prospects and challenges, and discuss the potential to directly correct genetic mutations in affected tissues and cells to treat diseases that are refractory to traditional therapies.
Therapeutic genome editing: prospects and challenges
TL;DR: Current progress toward developing programmable nuclease–based therapies as well as future prospects and challenges are discussed.
Journal ArticleDOI
HBV cccDNA: viral persistence reservoir and key obstacle for a cure of chronic hepatitis B
TL;DR: This review aims to summarise current knowledge on ccc DNA molecular biology, to highlight the experimental restrictions that have hitherto hampered faster progress and to discuss cccDNA as target for new, potentially curative therapies of chronic hepatitis B.
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Genome-editing Technologies for Gene and Cell Therapy
TL;DR: The mechanisms of different genome-editing strategies are presented and each of the common nuclease-based platforms, including zinc finger nucleases, transcription activator-like effector nucleases (TALENs), meganucleases, and the CRISPR/Cas9 system are described.
References
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Multiplex Genome Engineering Using CRISPR/Cas Systems
Le Cong,Le Cong,F. Ann Ran,F. Ann Ran,David M. Cox,David M. Cox,Shuailiang Lin,Shuailiang Lin,Robert P. J. Barretto,Naomi Habib,Patrick D. Hsu,Patrick D. Hsu,Xuebing Wu,Wenyan Jiang,Luciano A. Marraffini,Feng Zhang +15 more
TL;DR: The type II prokaryotic CRISPR (clustered regularly interspaced short palindromic repeats)/Cas adaptive immune system has been shown to facilitate RNA-guided site-specific DNA cleavage as discussed by the authors.
Image processing with ImageJ
TL;DR: ImageJ is an open source Java-written program that is used for many imaging applications, including those that that span the gamut from skin analysis to neuroscience, and can read most of the widely used and significant formats used in biomedical images.
Multiplex Genome Engineering Using CRISPR/Cas Systems
Le Cong,F. A. Ran,David Benjamin Turitz Cox,Shuailiang Lin,Robert P. J. Barretto,Naomi Habib,Patrick D. Hsu,Xuebing Wu,Wenyan Jiang,Luciano A. Marraffini,Feng Zhang +10 more
TL;DR: Two different type II CRISPR/Cas systems are engineered and it is demonstrated that Cas9 nucleases can be directed by short RNAs to induce precise cleavage at endogenous genomic loci in human and mouse cells, demonstrating easy programmability and wide applicability of the RNA-guided nuclease technology.
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High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells.
Yanfang Fu,Jennifer A Foden,Cyd Khayter,Morgan L. Maeder,Deepak Reyon,J. Keith Joung,Jeffry D. Sander +6 more
TL;DR: It is found that single and double mismatches are tolerated to varying degrees depending on their position along the guide RNA (gRNA)-DNA interface, and off-target cleavage of CRISPR-associated (Cas)9-based RGNs is characterized.
Journal ArticleDOI
Efficient genome editing in zebrafish using a CRISPR-Cas system
Woong Y. Hwang,Yanfang Fu,Deepak Reyon,Morgan L. Maeder,Shengdar Q. Tsai,Jeffry D. Sander,Randall T. Peterson,Randall T. Peterson,Jing-Ruey J. Yeh,J. Keith Joung +9 more
TL;DR: It is shown that the CRISPR-Cas system functions in vivo to induce targeted genetic modifications in zebrafish embryos with efficiencies similar to those obtained using zinc finger nucleases and transcription activator-like effector nucleases.