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Titlebook: Gene Correction; Methods and Protocol Francesca Storici Book 2014 Springer Science+Business Media, LLC 2014 gene correction.gene repair.gen

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Small Fragment Homologous Replacement (SFHR): Sequence-Specific Modification of Genomic DNA in Eukar atrophy, cystic fibrosis, severe combined immune deficiency). Several parameters can be modified to optimize the gene modification efficiency, as described in our recent study..In this chapter we describe the main guidelines that should be followed in SFHR application, in order to increase techniqu
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Preparation and Application of Triple Helix Forming Oligonucleotides and Single Strand Oligonucleotiactive psoralen-linked TFOs, their co-introduction into mammalian cells with donor nucleic acids, and the identification of cells with sequence conversion of the target site. We have emphasized details in the synthesis and purification of the oligonucleotides that are essential for preparation of re
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Lentiviral Vectors Encoding Zinc-Finger Nucleases Specific for the Model Target Locus ,rapid and simple restriction enzyme site polymorphism assay to detected DSB formation at the . target sequence. Owing in part to the small molecule-based clone selection schemes conferred by . allelic knockouts, this X-linked gene has become a “classical” target model locus in mammalian cells. The r
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CRISPR-Cas-Mediated Targeted Genome Editing in Human Cellshrough nonhomologous end joining (NHEJ) or through homologous recombination (HR) in the presence of an appropriate donor sequence. This RNA-guided editing tool offers greater ease of customization and synthesis in comparison to existing sequence-specific endonucleases and promises to become a highly
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Enhanced Gene Targeting of Adult and Pluripotent Stem Cells Using Evolved Adeno-associated Virus
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https://doi.org/10.1007/978-1-62703-761-7gene correction; gene repair; gene targeting; human genome; human pluripotent stem cells
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