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Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery

Article Abstract:

The infectivity of integrase-defective lentiviral vectors (IDLV) is used for expressing zinc-finger nucleases (ZFNs) and providing the template DNA for gene correction in different cell types. IDLV delivery and ZFNs targeting distinct loci are used for observing high levels of gene addition in a panel of human cell lines, as well as human embryonic stem cells, allowing rapid, selection-free isolation of clonogenic cells with the desired genetic modification.

Author: Naldini, Luigi, Galli, Cesare, Urnov, Fyodor D., Ya-Li Lee, Beausejour, Christian M., Gregory, Philip D., Holmes, Michael C., Kim, Kenneth A., Lombardo, Angelo, Genovese, Pietro, Colleoni, Silivia, Ando, Dale
Publisher: Nature Publishing Co.
Publication Name: Nature Biotechnology
Subject: Business
ISSN: 1087-0156
Year: 2007
Gene expression, Stem cell research

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Specificity by design

Article Abstract:

The specificity of a homing endonuclease is altered, using computational modeling of the protein-DNA interface. In the past few years there was great progress made in the design and selection of variant C2H2 zinc-finger proteins that can be targeted to be almost any desired site in the DNA.

Author: Pabo, Carl O.
Publisher: Nature Publishing Co.
Publication Name: Nature Biotechnology
Subject: Business
ISSN: 1087-0156
Year: 2006
Analysis, Genetic aspects, Cloning, Protein research, DNA-ligand interactions, DNA binding

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An improved zinc-finger nuclease architecture for highly specific genome editing

Article Abstract:

Two advanced zinc-finger nucleases using structure-based design which give high gene-modification efficiencies only when paired as a heterodimer with reduced levels of genome-wide cleavage are presented.

Author: Miller, Jeffrey C., Ya-Li Lee, Beausejour, Christian M., Holmes, Michael C., Pabo, Carl O., Wang, Jianbin, Guschin, Dmitry, Rupniewski, Igor, Waite, Adam J., Wang, Nathaniel S., Kim, Kenneth A., Gregory Philip D., Rebar, Edward J.
Publisher: Nature Publishing Co.
Publication Name: Nature Biotechnology
Subject: Business
ISSN: 1087-0156
Year: 2007
DNA repair

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Subjects list: Research, Zinc finger proteins, Usage, Nucleases
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