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
Reading frame correction by targeted genome editing restores dystrophin expression in cells from Duchenne muscular dystrophy patients.
David G. Ousterout,Pablo Perez-Pinera,Pratiksha I. Thakore,Ami M. Kabadi,Matthew T. Brown,Xiaoxia Qin,Olivier Fedrigo,Vincent Mouly,Jacques P. Tremblay,Charles A. Gersbach +9 more
TL;DR: It is shown that genome editing with transcription activator-like effector nucleases (TALENs), without a repair template, can efficiently correct the reading frame and restore the expression of a functional dystrophin protein that is mutated in DMD.
Journal ArticleDOI
Recent advances in engineering nonribosomal peptide assembly lines
TL;DR: The major advances made over the last decade in engineering the biosynthesis of nonribosomal peptides are reviewed and the new synthetic biology technologies promising to speed up the process are reviewed, enabling the creation and optimisation of many more assembly lines for heterologous expression.
Patent
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Zhang Feng,Le Cong,Fei Ran +2 more
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CRISPR/Cas9 for cancer research and therapy
TL;DR: Current applications of the CRISPR/Cas9 technology for cancer research and therapy are reviewed and novel Cas9 variants are described and how they are used in functional genomics to discover novel cancer-specific vulnerabilities are described.
Journal ArticleDOI
Poly peak parser: Method and software for identification of unknown indels using sanger sequencing of polymerase chain reaction products
TL;DR: Poly Peak Parser as mentioned in this paper is a tool that is able to separate ambiguous base calls into wild-type and mutant allele sequences, which can be used to identify heterozygous indels in many contexts.
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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