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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Journal ArticleDOI
Integrase deficient lentiviral vector: prospects for safe clinical applications
Chee-Hong Takahiro Yew,Narmatha Gurumoorthy,Fazlina Nordin,Gee Jun Tye,Wan Safwani Wan Kamarul Zaman,Jun Jie Tan,Min Hwei Ng +6 more
TL;DR: The generation of integrase-deficient lentiviral vectors by abrogating integrase activity in lentiv viral vector systems reduces the rate of transgenes integration into host genomes, and this feature is advantageous for therapeutic implementation and widens its clinical applications.
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CRISPR-Cas Systems: Prospects for Use in Medicine
TL;DR: The dCas9 system, based on a modified Cas9 devoid of nuclease activity, called CRISPRi, is widely used to control gene expression in bacteria for new drug biotargets validation and is also promising for therapy of genetic diseases.
Dissertation
Towards a Systematic Approach for Characterizing Regulatory Variation
TL;DR: This dissertation presents enhancer-FACSSeq, a high-throughput experimental approach for screening candidate enhancer sequences to test for in vivo, tissue-specific activity, and SIFTED, a statistical framework and web tool for the optimal design of TAL effectors, which have been used successfully in genome editing and can thus be used to test hypotheses about regulatory variants.
Journal ArticleDOI
DNA Detection Technology Using Zinc Finger Protein
TL;DR: It is suggested that detecting PCR products using double-stranded DNA (dsDNA) is more convenient and powerful compared with DNA probe hybridization technique.
Journal ArticleDOI
Donor T cells for CAR T cell therapy
Tiffany C Y Tang,Ning-Yi Xu,Robert E. Nordon,Michelle Haber,Kenneth P. Micklethwaite,Alla Dolnikov +5 more
TL;DR: In this paper , the authors used gene editing tools such as TALENs and CRISPR-Cas9 and non-gene editing methods such as short hairpin RNA and blockade of protein expression to enhance CAR T cell safety and efficacy by abrogating non-specific toxicity in the form of graft versus host disease.
References
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Journal ArticleDOI
Breaking the Code of DNA Binding Specificity of TAL-Type III Effectors
Jens Boch,Heidi Scholze,Sebastian Schornack,Angelika Landgraf,Simone Hahn,Sabine Kay,Thomas Lahaye,Anja Nickstadt,Ulla Bonas +8 more
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
Tomas Cermak,Erin L. Doyle,Michelle Christian,Li-Li Wang,Yong Zhang,Clarice Schmidt,Joshua A. Baller,Nikunj V. Somia,Adam J. Bogdanove,Daniel F. Voytas +9 more
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
Jeffrey C. Miller,Siyuan Tan,Guijuan Qiao,Kyle A. Barlow,Jianbin Wang,Danny F Xia,Xiangdong Meng,David Paschon,Elo Leung,Sarah J. Hinkley,Gladys P Dulay,Kevin Hua,Irina Ankoudinova,Gregory J. Cost,Fyodor D. Urnov,H. Steve Zhang,Michael C. Holmes,Lei Zhang,Philip D. Gregory,Edward J. Rebar +19 more
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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