A TALE nuclease architecture for efficient genome editing

@article{Miller2011ATN,
  title={A TALE nuclease architecture for efficient genome editing},
  author={Jeffrey C. Miller and Siyuan Tan and Guijuan Qiao and Kyle A. Barlow and Jianbin Wang and Danny F Xia and Xiangdong Meng and David E. Paschon and Elo Leung and Sarah Hinkley and Gladys P. Dulay and Kevin L Hua and Irina Ankoudinova and Gregory J Cost and Fyodor D. Urnov and H. Steve Zhang and Michael C. Holmes and Lei Zhang and Philip D. Gregory and Edward J Rebar},
  journal={Nature Biotechnology},
  year={2011},
  volume={29},
  pages={143-148}
}
Nucleases that cleave unique genomic sequences in living cells can be used for targeted gene editing and mutagenesis. [...] Key Result We identify TALE truncation variants that efficiently cleave DNA when linked to the catalytic domain of FokI and use these nucleases to generate discrete edits or small deletions within endogenous human NTF3 and CCR5 genes at efficiencies of up to 25%. We further show that designed TALEs can regulate endogenous mammalian genes. These studies demonstrate the effective application…Expand
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References

SHOWING 1-10 OF 53 REFERENCES
Critical parameters for genome editing using zinc finger nucleases.
TLDR
This review will discuss the advances in engineering custom zinc-finger nucleases and their application in stimulating homologous recombination in higher eukaryotic cells at efficiencies approaching 1 in 2 cells. Expand
Targeted genome editing in human cells with zinc finger nucleases constructed via modular assembly.
TLDR
This work synthesized and tested hundreds of ZFNs to target dozens of different sites in the human CCR5 gene-a co-receptor required for HIV infection-and found that many of these nucleases induced site-specific mutations in the C CR5 sequence. Expand
An improved zinc-finger nuclease architecture for highly specific genome editing
TLDR
Using structure-based design, two variant ZFNs are engineer that modify a native endogenous locus as efficiently as the parental architecture, but with a >40-fold reduction in homodimer function and much lower levels of genome-wide cleavage. Expand
Genome editing with engineered zinc finger nucleases
TLDR
A broad range of outcomes has resulted from the application of the same core technology: targeted genome cleavage by engineered, sequence-specific zinc finger nucleases followed by gene modification during subsequent repair. Expand
Targeting DNA Double-Strand Breaks with TAL Effector Nucleases
TLDR
A new class of sequence-specific nucleases created by fusing transcription activator-like effectors (TALEs) to the catalytic domain of the FokI endonuclease is reported. Expand
Enhancing zinc-finger-nuclease activity with improved obligate heterodimeric architectures
TLDR
The development and application of a yeast-based selection system designed to functionally interrogate the ZFN dimer interface is reported, identified critical residues involved in dimerization through the isolation of cold-sensitive nuclease domains, and used to engineer ZFNs that have superior cleavage activity while suppressing homodimerization. Expand
Zinc Finger Nucleases as tools to understand and treat human diseases
TLDR
This review discusses the development and use of designer zinc finger proteins (ZFPs) as sequence specific tools, and the utility of other functional domains, such as transcriptional activators and repressors, and highlights how these are being used as discovery and therapeutic tools. Expand
TAL nucleases (TALNs): hybrid proteins composed of TAL effectors and FokI DNA-cleavage domain
TLDR
The creation and initial characterization of a group of rare-cutting, site-specific DNA nucleases produced by fusion of the restriction enzyme FokI endonuclease domain (FN) with the high-specificity DNA-binding domains of AvrXa7 and PthXo1 are reported. Expand
Zinc-finger nucleases: the next generation emerges.
  • T. Cathomen, J. Joung
  • Biology, Medicine
  • Molecular therapy : the journal of the American Society of Gene Therapy
  • 2008
TLDR
Recent advances in gene targeting, and upcoming challenges for, this emerging technology are reviewed and future experimental work that will be needed to bring ZFNs safely into a clinical setting are discussed. Expand
A rapid and general assay for monitoring endogenous gene modification.
TLDR
A simple procedure for quantifying mutations that result from DNA double-strand break repair via non-homologous end joining based on the ability of the Surveyor nuclease to selectively cleave distorted duplex DNA formed via cross-annealing of mutated and wild-type sequence is described. Expand
...
1
2
3
4
5
...