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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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Gene-based therapies for neurodegenerative diseases.

TL;DR: The recent success of a viral-vector-based gene therapy in spinal muscular atrophy-promoting survival and motor function with a single intravenous injection-offers a paradigm for such therapeutic intervention and a platform to build on as discussed by the authors.
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Engineering synthetic TALE and CRISPR/Cas9 transcription factors for regulating gene expression

TL;DR: Two platforms for designing synthetic transcription factors for manipulating gene expression are reviewed: Transcription activator-like effectors (TALEs) and the RNA-guided clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 system.
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Building a Safer and Faster CAR: Seatbelts, Airbags, and CRISPR.

TL;DR: The recent report of a nonhuman primate model for CRS is poised to help advance the management of this syndrome and new engineering modalities, based on the use of targeted nucleases like CRISPR, may further enhance the efficacy and safety of CAR T cells.
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Genetically engineered microbial remediation of soils co-contaminated by heavy metals and polycyclic aromatic hydrocarbons: Advances and ecological risk assessment.

TL;DR: In this paper, a review of successful bioremediation techniques and approaches for soil contaminated with heavy metals and/or polycyclic aromatic hydrocarbons by GEMs, and discusses some challenges in the simultaneous removal of HMs and PAHs from soil by designing multi-functional genetic engineering microorganisms.
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A Mitocentric View of Alzheimer’s Disease

TL;DR: This article reviews the previous related studies from different aspects and concludes the critical roles of mitochondrial dysfunction in AD, suggesting a different route to AD therapy, which may guide the research and treatment direction.
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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