Development of an intein-mediated split–Cas9 system for gene therapy
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Wolfgang Wurst | Stefan Engelhardt | Ralf Kühn | W. Wurst | Ralf Kühn | S. Engelhardt | Oskar Ortiz | Oskar Ortiz | Dong-Jiunn Jeffery Truong | Karin Kühner | Stanislas Werfel | Stanislas Werfel | Karin Kühner
[1] Kira S. Makarova,et al. Phylogeny of Cas9 determines functional exchangeability of dual-RNA and Cas9 among orthologous type II CRISPR-Cas systems , 2013, Nucleic acids research.
[2] Chandra Sekhar Pedamallu,et al. Faster Protein Splicing with the Nostoc punctiforme DnaE Intein Using Non-native Extein Residues , 2013, The Journal of Biological Chemistry.
[3] W. Wurst,et al. Highly Efficient Targeted Mutagenesis in Mice Using TALENs , 2013, Genetics.
[4] Nicholas E. Propson,et al. Efficient genome engineering in human pluripotent stem cells using Cas9 from Neisseria meningitidis , 2013, Proceedings of the National Academy of Sciences.
[5] Randall J. Platt,et al. Optical Control of Mammalian Endogenous Transcription and Epigenetic States , 2013, Nature.
[6] B. Wang,et al. Protein trans-splicing as a means for viral vector-mediated in vivo gene therapy. , 2008, Human Gene Therapy.
[7] Jennifer A. Doudna,et al. Structures of Cas9 Endonucleases Reveal RNA-Mediated Conformational Activation , 2014, Science.
[8] Feng Zhang,et al. A split-Cas9 architecture for inducible genome editing and transcription modulation , 2015, Nature Biotechnology.
[9] Benjamin L. Oakes,et al. Programmable RNA recognition and cleavage by CRISPR/Cas9 , 2014, Nature.
[10] Christopher A. Voigt,et al. Principles of genetic circuit design , 2014, Nature Methods.
[11] Jeffrey J. Tabor,et al. Post-translational tools expand the scope of synthetic biology. , 2012, Current opinion in chemical biology.
[12] M. Ehlers,et al. Optimizing promoters for recombinant adeno-associated virus-mediated gene expression in the peripheral and central nervous system using self-complementary vectors. , 2011, Human gene therapy.
[13] Amy B Tyszkiewicz,et al. Activation of protein splicing with light in yeast , 2008, Nature Methods.
[14] W. Wurst,et al. Creation of targeted genomic deletions using TALEN or CRISPR/Cas nuclease pairs in one-cell mouse embryos , 2014, FEBS open bio.
[15] Prashant Mali,et al. Orthogonal Cas9 Proteins for RNA-Guided Gene Regulation and Editing , 2013, Nature Methods.
[16] L. Vandenberghe,et al. Adeno-associated virus: fit to serve. , 2014, Current opinion in virology.
[17] W. Wurst,et al. Reversible and tissue‐specific activation of MAP kinase signaling by tamoxifen in brafV637ERT2 mice , 2013, Genesis.
[18] T. Muir,et al. Kinetic control of one-pot trans-splicing reactions by using a wild-type and designed split intein. , 2011, Angewandte Chemie.
[19] S. Mittal,et al. Adenoviral vector immunity: its implications and circumvention strategies. , 2011, Current gene therapy.
[20] K. Dill. Theory for the folding and stability of globular proteins. , 1985, Biochemistry.
[21] J. Doudna,et al. A Programmable Dual-RNA–Guided DNA Endonuclease in Adaptive Bacterial Immunity , 2012, Science.
[22] T. Muir,et al. Protein splicing triggered by a small molecule. , 2002, Journal of the American Chemical Society.
[23] M. Isalan,et al. A split intein T7 RNA polymerase for transcriptional AND-logic , 2014, Nucleic acids research.
[24] Luke A. Gilbert,et al. CRISPR-Mediated Modular RNA-Guided Regulation of Transcription in Eukaryotes , 2013, Cell.
[25] J. Keith Joung,et al. FLASH Assembly of TALENs Enables High-Throughput Genome Editing , 2012, Nature Biotechnology.
[26] Daniel J. Rader,et al. Permanent Alteration of PCSK9 With In Vivo CRISPR-Cas9 Genome Editing , 2014, Circulation research.
[27] Ryan R. Richardson,et al. Two- and three-input TALE-based AND logic computation in embryonic stem cells , 2013, Nucleic acids research.
[28] Yarden Katz,et al. Multiplexed activation of endogenous genes by CRISPR-on, an RNA-guided transcriptional activator system , 2013, Cell Research.
[29] Dominik Niopek,et al. CRISPR/Cas9-mediated genome engineering: an adeno-associated viral (AAV) vector toolbox. , 2014, Biotechnology journal.
[30] Ulrike Winkler,et al. Split-CreERT2: Temporal Control of DNA Recombination Mediated by Split-Cre Protein Fragment Complementation , 2009, PloS one.
[31] Tom W Muir,et al. Post-translational enzyme activation in an animal via optimized conditional protein splicing. , 2007, Nature chemical biology.
[32] Hao Yin,et al. Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype , 2014, Nature Biotechnology.
[33] V. Iyer,et al. Efficient genome modification by CRISPR-Cas9 nickase with minimal off-target effects , 2014, Nature Methods.
[34] L A Herzenberg,et al. Disruption of overlapping transcripts in the ROSA beta geo 26 gene trap strain leads to widespread expression of beta-galactosidase in mouse embryos and hematopoietic cells. , 1997, Proceedings of the National Academy of Sciences of the United States of America.
[35] Ethan Bier,et al. The mutagenic chain reaction: A method for converting heterozygous to homozygous mutations , 2015, Science.
[36] F. Mingozzi,et al. Cell-Mediated Immunity to AAV Vectors, Evolving Concepts and Potential Solutions , 2014, Front. Immunol..
[37] David Bryder,et al. Efficient ablation of genes in human hematopoietic stem and effector cells using CRISPR/Cas9. , 2014, Cell stem cell.
[38] Samuel H Sternberg,et al. Rational design of a split-Cas9 enzyme complex , 2015, Proceedings of the National Academy of Sciences.
[39] David A. Scott,et al. In vivo genome editing using Staphylococcus aureus Cas9 , 2015, Nature.
[40] W. Wurst,et al. Modeling disease mutations by gene targeting in one-cell mouse embryos , 2012, Proceedings of the National Academy of Sciences.
[41] A. Nordheim,et al. Rapid and highly efficient inducible cardiac gene knockout in adult mice using AAV-mediated expression of Cre recombinase. , 2014, Cardiovascular research.
[42] P. Lyu,et al. Circular Permutation Prediction Reveals a Viable Backbone Disconnection for Split Proteins: An Approach in Identifying a New Functional Split Intein , 2012, PloS one.
[43] Jeffry D Sander,et al. FLAsH assembly of TALeNs for high-throughput genome editing , 2022 .
[44] Feng Zhang,et al. Crystal Structure of Cas9 in Complex with Guide RNA and Target DNA , 2014, Cell.