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:Â
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
Gene editing for inflammatory disorders.
TL;DR: The ability to orchestrate precise targeted modifications to the genome will likely enable cell-based therapies for inflammatory diseases such as monogenic autoinflammatory disease, acquired autoimmune disease and for regenerative medicine in the setting of an inflammatory environment.
Journal ArticleDOI
Models and strategies in the development of antiobesity drugs.
Amy Agahi,Kevin Murphy +1 more
TL;DR: The physiological basis of appetite, rodent models used in the development of anti-obesity drugs, and potential future targets for novel antiobesity agents are described.
Journal ArticleDOI
TALENs-mediated gene disruption of FLT3 in leukemia cells: Using genome-editing approach for exploring the molecular basis of gene abnormality.
Jue Wang,Tongjuan Li,Mi Zhou,Zheng Hu,Xiaoxi Zhou,Shiqiu Zhou,Na Wang,Liang Huang,Lei Zhao,Yang Cao,Min Xiao,Ding Ma,Pengfei Zhou,Zhen Shang,Jianfeng Zhou +14 more
TL;DR: The data support that this genome-editing approach is a robust and generally applicable platform to explore the molecular bases of gene mutations and an activation of nonsense-mediated decay pathway in mutant K562 clones as well as an inhibited FLT3 signaling.
Journal ArticleDOI
Ctrl-Alt-inDel: genome editing to reprogram a cell in the clinic.
TL;DR: Three ongoing developments will ensure that the range of edited and reprogrammed cells to enter the clinic, and the scope of target indications, will grow markedly in the next five years: the ability to identify disease-relevant targets in noncoding regulatory DNA, which is uniquely suited for editing-based cell program control; recent reduction to clinical practice of in vivo editing; and progress in engineering and manufacture of differentiated cells from pluripotent progenitors.
Journal ArticleDOI
Identification of organ-autonomous constituents of the molecular memory conferred by thyroid hormone exposure in cold temperature-arrested metamorphosing Rana (Lithobates) catesbeiana tadpoles
TL;DR: It is determined that thibz is the only transcript that is TH-responsive after 2 days post-exposure at low temperature in both tissues in the intact animal, and endogenous factors that modulate the molecular memory in intact animals are identified.
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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