TALENs: a widely applicable technology for targeted genome editing
J. Keith Joung,Jeffry D. Sander +1 more
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TLDR
The newly-developed transcription activator-like effector nucleases (TALENs) comprise a nonspecific DNA-cleaving nuclease fused to a DNA-binding domain that can be easily engineered so that TALens can target essentially any sequence.Abstract:
Engineered nucleases enable the targeted alteration of nearly any gene in a wide range of cell types and organisms. The newly-developed transcription activator-like effector nucleases (TALENs) comprise a nonspecific DNA-cleaving nuclease fused to a DNA-binding domain that can be easily engineered so that TALENs can target essentially any sequence. The capability to quickly and efficiently alter genes using TALENs promises to have profound impacts on biological research and to yield potential therapeutic strategies for genetic diseases.read more
Citations
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CRISPR-Mediated Modular RNA-Guided Regulation of Transcription in Eukaryotes
Luke A. Gilbert,Matthew H. Larson,Leonardo Morsut,Zairan Liu,Gloria A. Brar,Sandra Elizabeth Torres,Noam Stern-Ginossar,Onn Brandman,Evan H. Whitehead,Jennifer A. Doudna,Wendell A. Lim,Jonathan S. Weissman,Lei S. Qi +12 more
TL;DR: The results establish that the CRISPR system can be used as a modular and flexible DNA-binding platform for the recruitment of proteins to a target DNA sequence, revealing the potential of CRISpri as a general tool for the precise regulation of gene expression in eukaryotic cells.
Journal ArticleDOI
CRISPR-Cas systems for editing, regulating and targeting genomes
Jeffry D. Sander,J. Keith Joung +1 more
TL;DR: A modified version of the CRISPR-Cas9 system has been developed to recruit heterologous domains that can regulate endogenous gene expression or label specific genomic loci in living cells, which will undoubtedly transform biological research and spur the development of novel molecular therapeutics for human disease.
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.
Journal ArticleDOI
Efficient multiplex biallelic zebrafish genome editing using a CRISPR nuclease system
TL;DR: An improved CRISPR/Cas system in zebra fish with custom guide RNAs and a zebrafish codon-optimized Cas9 protein that efficiently targeted a reporter transgene Tg(-5.1mnx1:egfp) and four endogenous loci and five genomic loci, resulting in multiple loss-of-function phenotypes in the same injected fish.
Journal ArticleDOI
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.
References
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Journal ArticleDOI
Heritable targeted gene disruption in zebrafish using designed zinc-finger nucleases
Yannick Doyon,Jasmine M. McCammon,Jeffrey C. Miller,Farhoud Faraji,Catherine Ngo,George E. Katibah,Rainier Amora,Toby Dylan Hocking,Lei Zhang,Edward J. Rebar,Philip D. Gregory,Fyodor D. Urnov,Fyodor D. Urnov,Sharon L. Amacher +13 more
TL;DR: The frequency and precision of gene-disruption events observed suggest that this approach should be applicable to any loci in zebrafish or in other organisms that allow mRNA delivery into the fertilized egg.
Journal ArticleDOI
Xanthomonas AvrBs3 Family-Type III Effectors: Discovery and Function
Jens Boch,Ulla Bonas +1 more
TL;DR: The discovery of TAL effectors is described, which act as transcriptional activators in the plant cell nucleus and are determined by a novel modular DNA-binding domain.
Journal ArticleDOI
Efficient construction of sequence-specific TAL effectors for modulating mammalian transcription
Feng Zhang,Le Cong,Le Cong,Simona Lodato,Sriram Kosuri,Sriram Kosuri,George M. Church,George M. Church,Paola Arlotta +8 more
TL;DR: 17 TALEs are synthesized that are customized to recognize specific DNA-binding sites, and it is demonstrated that they can specifically modulate transcription of endogenous genes (SOX2 and KLF4) in human cells.
Journal ArticleDOI
Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery
Angelo Lombardo,Angelo Lombardo,Pietro Genovese,Christian Beauséjour,Silvia Colleoni,Silvia Colleoni,Ya Li Lee,Kenneth Kim,Dale Ando,Fyodor D. Urnov,Cesare Galli,Philip D. Gregory,Michael C. Holmes,Luigi Naldini +13 more
TL;DR: Using IDLV delivery and ZFNs targeting distinct loci, high levels of gene addition are observed in a panel of human cell lines, as well as human embryonic stem cells, allowing rapid, selection-free isolation of clonogenic cells with the desired genetic modification.
Journal ArticleDOI
High-efficiency TALEN-based gene editing produces disease-resistant rice
TL;DR: TALEN technology is exploited to edit a specific S gene in rice to thwart the virulence strategy of X. oryzae and thereby engineer heritable genome modifications for resistance to bacterial blight, a devastating disease in a crop that feeds half of the world’s population.
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