ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering.

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Published in Trends Biotechnol on May 09, 2013

Authors

Thomas Gaj1, Charles A Gersbach, Carlos F Barbas

Author Affiliations

1: The Skaggs Institute for Chemical Biology, The Scripps Research Institute, La Jolla, CA, USA.

Associated clinical trials:

Autologous T-Cells Genetically Modified at the CCR5 Gene by Zinc Finger Nucleases SB-728 for HIV (Zinc-Finger) | NCT00842634

Phase 1 Dose Escalation Study of Autologous T-cells Genetically Modified at the CCR5 Gene by Zinc Finger Nucleases in HIV-Infected Patients | NCT01044654

Study of Autologous T-cells Genetically Modified at the CCR5 Gene by Zinc Finger Nucleases in HIV-Infected Subjects | NCT01252641

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Synthetic zinc finger proteins: the advent of targeted gene regulation and genome modification technologies. Acc Chem Res (2014) 1.06

{gamma}-Globin gene expression in chemical inducer of dimerization (CID)-dependent multipotential cells established from human {beta}-globin locus yeast artificial chromosome ({beta}-YAC) transgenic mice. J Biol Chem (2005) 1.06

A comprehensive approach to zinc-finger recombinase customization enables genomic targeting in human cells. Nucleic Acids Res (2013) 1.04

Phenotypic knockout of VEGF-R2 and Tie-2 with an intradiabody reduces tumor growth and angiogenesis in vivo. Proc Natl Acad Sci U S A (2005) 1.04

Synthesis of the next-generation therapeutic antibodies that combine cell targeting and antibody-catalyzed prodrug activation. Proc Natl Acad Sci U S A (2007) 1.04

A highly enantioselective amino acid-catalyzed route to functionalized alpha-amino acids. J Am Chem Soc (2002) 1.03

A highly enantioselective route to either enantiomer of both alpha- and beta-amino acid derivatives. J Am Chem Soc (2002) 1.03

Beta-lactam-based approach for the chemical programming of aldolase antibody 38C2. Bioorg Med Chem Lett (2009) 1.03

The direct organocatalytic asymmetric mannich reaction: unmodified aldehydes as nucleophiles. J Org Chem (2003) 1.02

In vivo selection of combinatorial libraries and designed affinity maturation of polydactyl zinc finger transcription factors for ICAM-1 provides new insights into gene regulation. J Mol Biol (2004) 1.02

Virus-based gene therapy strategies for bone regeneration. Biomaterials (2006) 1.01

Development of a small peptide tag for covalent labeling of proteins. Bioconjug Chem (2007) 1.00

Synthesis of beta-hydroxyaldehydes with stereogenic quaternary carbon centers by direct organocatalytic asymmetric aldol reactions. Angew Chem Int Ed Engl (2004) 1.00

Human/mouse cross-reactive anti-VEGF receptor 2 recombinant antibodies selected from an immune b9 allotype rabbit antibody library. J Immunol Methods (2004) 1.00

Design and use of fluorogenic aldehydes for monitoring the progress of aldehyde transformations. J Am Chem Soc (2004) 0.99

Promoter-targeted phage display selections with preassembled synthetic zinc finger libraries for endogenous gene regulation. J Mol Biol (2004) 0.98

Direct asymmetric anti-Mannich-type reactions catalyzed by a designed amino acid. J Am Chem Soc (2006) 0.98

Thiourea-catalyzed highly enantio- and diastereoselective additions of oxindoles to nitroolefins: application to the formal synthesis of (+)-physostigmine. J Am Chem Soc (2009) 0.97

Controlling gene expression in plants using synthetic zinc finger transcription factors. Plant J (2002) 0.97

Highly enantio- and diastereoselective Mannich reactions of glycine Schiff bases with in situ generated N-Boc-imines catalyzed by a cinchona alkaloid thiourea. Org Lett (2010) 0.96

Proline-catalyzed one-step asymmetric synthesis of 5-hydroxy-(2E)-hexenal from acetaldehyde. J Org Chem (2002) 0.96

Regulation of transgene expression in plants with polydactyl zinc finger transcription factors. Proc Natl Acad Sci U S A (2002) 0.96

Expanding the concept of chemically programmable antibodies to RNA aptamers: chemically programmed biotherapeutics. Angew Chem Int Ed Engl (2010) 0.95

Construction of bispirooxindoles containing three quaternary stereocentres in a cascade using a single multifunctional organocatalyst. Nat Chem (2011) 0.95

Targeting tumor angiogenesis with adenovirus-delivered anti-Tie-2 intrabody. Cancer Res (2005) 0.95

Organocatalytic direct asymmetric aldol reactions in water. J Am Chem Soc (2006) 0.95

Regulation of endogenous human gene expression by ligand-inducible TALE transcription factors. ACS Synth Biol (2013) 0.94

Catalysis of 3-pyrrolidinecarboxylic acid and related pyrrolidine derivatives in enantioselective anti-Mannich-type reactions: importance of the 3-acid group on pyrrolidine for stereocontrol. J Am Chem Soc (2007) 0.94

Small-molecule switches for zinc finger transcription factors. J Am Chem Soc (2003) 0.94

Direct observation of an enamine intermediate in amine catalysis. J Am Chem Soc (2009) 0.94

Monoclonal antibody therapeutics with up to five specificities: functional enhancement through fusion of target-specific peptides. MAbs (2013) 0.93

Improving the serum stability of site-specific antibody conjugates with sulfone linkers. Bioconjug Chem (2014) 0.93

Direct asymmetric organocatalytic Michael reactions of alpha,alpha-disubstituted aldehydes with beta-nitrostyrenes for the synthesis of quaternary carbon-containing products. Org Lett (2004) 0.92

Isolation of human prostate cancer cell reactive antibodies using phage display technology. J Immunol Methods (2004) 0.92

Asymmetric synthesis of quaternary alpha- and beta-amino acids and beta-lactams via proline-catalyzed mannich reactions with branched aldehyde donors. Org Lett (2004) 0.92