ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering
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TLDR
A review of achievements made possible by site-specific nuclease technologies and applications of these reagents for genetic analysis and manipulation, including the therapeutic potential of ZFNs and TALENs, and future prospects for the field are discussed.About:
This article is published in Trends in Biotechnology.The article was published on 2013-07-01 and is currently open access. It has received 3235 citations till now. The article focuses on the topics: Genome editing & Transcription activator-like effector nuclease.read more
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Journal ArticleDOI
Non-viral vectors for gene-based therapy
Hao Yin,Rosemary Lynn Kanasty,Ahmed A. Eltoukhy,Arturo J. Vegas,J. Robert Dorkin,Daniel G. Anderson +5 more
TL;DR: The biological barriers to gene delivery in vivo are introduced and recent advances in material sciences, nanotechnology and nucleic acid chemistry that have yielded promising non-viral delivery systems are discussed, some of which are currently undergoing testing in clinical trials.
Journal ArticleDOI
Genome-Scale CRISPR-Mediated Control of Gene Repression and Activation
Luke A. Gilbert,Max A. Horlbeck,Britt Adamson,Jacqueline E. Villalta,Yuwen Chen,Evan H. Whitehead,Carla P. Guimaraes,Barbara Panning,Hidde L. Ploegh,Michael C. Bassik,Lei S. Qi,Martin Kampmann,Jonathan S. Weissman +12 more
TL;DR: This work identifies rules for specific targeting of transcriptional repressors (CRISPRi), typically achieving 90%-99% knockdown with minimal off-target effects, and activators to endogenous genes via endonuclease-deficient Cas9, which enable modulation of gene expression over a ∼1,000-fold range.
Journal ArticleDOI
Easy quantitative assessment of genome editing by sequence trace decomposition
TL;DR: TIDE, a method that requires only a pair of PCR reactions and two standard capillary sequencing runs to identify the major induced mutations in the projected editing site and accurately determines their frequency in a cell population, is presented.
Journal ArticleDOI
Dynamic Imaging of Genomic Loci in Living Human Cells by an Optimized CRISPR/Cas System
Baohui Chen,Luke A. Gilbert,Beth A. Cimini,Joerg Schnitzbauer,Wei Zhang,Gene-Wei Li,Jason Park,Elizabeth H. Blackburn,Jonathan S. Weissman,Lei S. Qi,Bo Huang +10 more
TL;DR: Using an EGFP-tagged endonuclease-deficient Cas9 protein and a structurally optimized small guide (sg) RNA, robust imaging of repetitive elements in telomeres and coding genes in living cells is demonstrated by repurposing the bacterial CRISPR/Cas system.
Genome-Scale CRISPR-Mediated Control of Gene Repression and Activation
Luke A. Gilbert,Max A. Horlbeck,Britt Adamson,Jacqueline E. Villalta,Yuwen Chen,Evan H. Whitehead,Carla P. Guimaraes,Barbara Panning,Michael C. Bassik,Lei S. Qi,Martin Kampmann,Jonathan S. Weissman,Hidde L. Ploegh +12 more
TL;DR: In this article, the authors identify rules for specific targeting of transcriptional repressors (CRISPRi), typically achieving 90%-99% knockdown with minimal off-target effects, and activators (CRisPRa) to endogenous genes via endonuclease-deficient Cas9.
References
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Journal ArticleDOI
An integrated encyclopedia of DNA elements in the human genome
TL;DR: The Encyclopedia of DNA Elements project provides new insights into the organization and regulation of the authors' genes and genome, and is an expansive resource of functional annotations for biomedical research.
Journal ArticleDOI
A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity.
Martin Jinek,Krzysztof Chylinski,Krzysztof Chylinski,Ines Fonfara,Michael H. Hauer,Jennifer A. Doudna,Emmanuelle Charpentier +6 more
TL;DR: This study reveals a family of endonucleases that use dual-RNAs for site-specific DNA cleavage and highlights the potential to exploit the system for RNA-programmable genome editing.
Journal ArticleDOI
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.
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.
Journal ArticleDOI
RNA-Guided Human Genome Engineering via Cas9
Prashant Mali,Luhan Yang,Kevin M. Esvelt,John Aach,Marc Güell,James E. DiCarlo,Julie E. Norville,George M. Church,George M. Church +8 more
TL;DR: The type II bacterial CRISPR system is engineer to function with custom guide RNA (gRNA) in human cells to establish an RNA-guided editing tool for facile, robust, and multiplexable human genome engineering.
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Patrick D. Hsu,David A. Scott,David A. Scott,Joshua A. Weinstein,Joshua A. Weinstein,F. Ann Ran,F. Ann Ran,F. Ann Ran,Silvana Konermann,Silvana Konermann,Vineeta Agarwala,Vineeta Agarwala,Vineeta Agarwala,Yinqing Li,Yinqing Li,Eli J. Fine,Xuebing Wu,Ophir Shalem,Ophir Shalem,Thomas J. Cradick,Luciano A. Marraffini,Gang Bao,Feng Zhang,Feng Zhang +23 more