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Open AccessJournal ArticleDOI

TALENs: a widely applicable technology for targeted genome editing

J. Keith Joung, +1 more
- 01 Jan 2013 - 
- Vol. 14, Iss: 1, pp 49-55
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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.

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Citations
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Functional interrogation of non-coding DNA through CRISPR genome editing.

TL;DR: CRISPR-based loss- and gain-of-function techniques for the interrogation of non-coding DNA are reviewed.
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Progress in gene therapy for primary immunodeficiencies using lentiviral vectors.

TL;DR: Substantial progress has been made to make lentiviral gene therapy platforms available for a number of rare genetic diseases, with main focus on Wiskott–Aldrich syndrome, chronic granulomatous disease and adenosine deaminase deficient severe combined immunodeficiency.
Journal ArticleDOI

Simultaneous Gene Editing by Injection of mRNAs Encoding Transcription Activator-Like Effector Nucleases into Mouse Zygotes

TL;DR: Injection of TALEN mRNAs into mouse zygotes transferred into foster mothers efficiently generated founder mice with heritable mutations in targeted genes, bypassing constraints of conventional mouse knockout technology in embryonic stem cells.
Journal ArticleDOI

CRISPR-Cas9: from Genome Editing to Cancer Research.

TL;DR: Recent technical advances in the application of CRISPR-Cas9 system in cancer genetics, large-scale cancer driver gene hunting, animal cancer modeling and functional studies are viewed.
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Application of CRISPR/Cas9 gene editing technique in the study of cancer treatment.

TL;DR: The findings of CRISPR/Cas9 for tumor treatment research are reviewed to provide references for related future studies on the pathogenesis and clinical treatment of tumors.
References
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Journal ArticleDOI

Breaking the Code of DNA Binding Specificity of TAL-Type III Effectors

TL;DR: The functionality of a distinct type of DNA binding domain is described and allows the design ofDNA binding domains for biotechnology.
Journal ArticleDOI

Efficient design and assembly of custom TALEN and other TAL effector-based constructs for DNA targeting

TL;DR: A method and reagents for efficiently assembling TALEN constructs with custom repeat arrays are presented and design guidelines based on naturally occurring TAL effectors and their binding sites are described.
Journal ArticleDOI

A TALE nuclease architecture for efficient genome editing

TL;DR: This study identifies TALE truncation variants that efficiently cleave DNA when linked to the catalytic domain of FokI and uses them to generate discrete edits or small deletions within endogenous human NTF3 and CCR5 genes at efficiencies of up to 25%.
Journal ArticleDOI

Genome editing with engineered zinc finger nucleases

TL;DR: 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.
Journal ArticleDOI

A Simple Cipher Governs DNA Recognition by TAL Effectors

TL;DR: It is shown that a repeat-variable pair of residues specifies the nucleotides in the target site, one pair to one nucleotide, with no apparent context dependence, which represents a previously unknown mechanism for protein-DNA recognition that explains TAL effector specificity, enables target site prediction, and opens prospects for use of TAL effects in research and biotechnology.
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