TALEN-based Gene Correction for Epidermolysis Bullosa
Mark J. Osborn,Colby G. Starker,Amber N. McElroy,Beau R. Webber,Megan J. Riddle,Lily Xia,Anthony P. DeFeo,Richard Gabriel,Manfred Schmidt,Christof von Kalle,Daniel F. Carlson,Morgan L. Maeder,J. Keith Joung,John E. Wagner,Daniel F. Voytas,Bruce R. Blazar,Jakub Tolar +16 more
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TLDR
This study provides proof-of-concept for TALen-mediated in situ correction of an endogenous patient-specific gene mutation and used an unbiased screen for comprehensive TALEN target mapping that will cooperatively facilitate translational application.About:
This article is published in Molecular Therapy.The article was published on 2013-06-01 and is currently open access. It has received 286 citations till now. The article focuses on the topics: Transcription activator-like effector nuclease & Gene mutation.read more
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GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases
Shengdar Q. Tsai,Zongli Zheng,Nhu T. Nguyen,Matthew Liebers,Ved V Topkar,Vishal Thapar,Nicolas Wyvekens,Cyd Khayter,A. John Iafrate,Long P. Le,Martin J. Aryee,J. Keith Joung +11 more
TL;DR: The experiments define the most rigorous framework for genome-wide identification of RGN off-target effects to date and provide a method for evaluating the safety of these nucleases before clinical use.
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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
Off-target Effects in CRISPR/Cas9-mediated Genome Engineering
TL;DR: The improvement off-target specificity in the CRISPR/Cas9 system will provide solid genotype–phenotype correlations, and thus enable faithful interpretation of genome-editing data, which will certainly facilitate the basic and clinical application of this technology.
Journal ArticleDOI
Prevention of muscular dystrophy in mice by CRISPR/Cas9–mediated editing of germline DNA
Chengzu Long,John R. McAnally,John M. Shelton,Alex A. Mireault,Rhonda Bassel-Duby,Eric N. Olson +5 more
TL;DR: A mutation that causes muscular dystrophy in mice can be corrected by genome editing, which prevents the disease from developing, and this proof of concept sets the stage for applying genome editing to specific cell types involved in the disease.
Journal ArticleDOI
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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Zinc fingers hit off target
TL;DR: ZFNs, in addition to cleaving at their desired sites, can also have unexpected cleavage effects in vivo that cannot be predicted using conventional in silico analyses, which could have important consequences for the safe use and optimization of ZFNs in gene therapy.