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Distinct Factors Control Histone Variant H3.3 Localization at Specific Genomic Regions
TLDR
It is demonstrated that multiple and distinct factors are responsible for H3.3 localization at specific genomic locations in mammalian cells. Expand
Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases
TLDR
The highly efficient targeting of three genes in human pluripotent cells using zinc-finger nuclease (ZFN)–mediated genome editing is reported, demonstrating that ZFNs can be used to generate reporter cells by targeting non-expressed genes in hESCs and hiPSCs. Expand
Precise genome modification in the crop species Zea mays using zinc-finger nucleases
TLDR
ZFNs can be used in any plant species amenable to DNA delivery and are established as a new strategy for plant genetic manipulation in basic science and agricultural applications. Expand
Targeted gene addition into a specified location in the human genome using designed zinc finger nucleases
TLDR
Data reveal the surprising versatility of the specialized polymerase machinery involved in double-strand break repair, illuminate a powerful approach to mammalian cell engineering, and open the possibility of ZFN-driven gene addition therapy for human genetic disease. Expand
SON controls cell-cycle progression by coordinated regulation of RNA splicing.
TLDR
It is reported that SON, a large Ser/Arg (SR)-related protein, is a splicing cofactor contributing to efficient splicing of cell-cycle regulators, suggesting its function in efficient cotranscriptional RNA processing. Expand
Functional genomics, proteomics, and regulatory DNA analysis in isogenic settings using zinc finger nuclease-driven transgenesis into a safe harbor locus in the human genome.
TLDR
The use of designed zinc finger nucleases (ZFNs) for efficient transgenesis without drug selection into the PPP1R12C gene, a "safe harbor" locus known as AAVS1, allows bona fide isogenic settings for high-throughput functional genomics, proteomics, and regulatory DNA analysis in essentially any transformed human cell type and in primary cells. Expand
In vivo genome editing of the albumin locus as a platform for protein replacement therapy.
TLDR
A general strategy for liver-directed protein replacement therapies that addresses site-specific integration of therapeutic transgenes within the albumin gene and the establishment of a universal nuclease-based platform for secreted protein production is reported. Expand
Targeted transgene integration in plant cells using designed zinc finger nucleases
TLDR
It is reported that designed zinc finger nucleases (ZFNs) can drive site-directed DNA integration into transgenic and native gene loci and point toward a novel approach for targeted gene addition, replacement and trait stacking in plants. Expand
Zinc-finger nuclease-driven targeted integration into mammalian genomes using donors with limited chromosomal homology
TLDR
It is demonstrated that easily-generated linear donors with extremely short homology regions drive transgene integration into 5–10% of chromosomes and that oligonucleotide donors with single-stranded 5′ overhangs complementary to those made by ZFNs are efficiently ligated in vivo to the DSB. Expand
Chromosomal translocations induced at specified loci in human stem cells
TLDR
The approach described in this report is readily applicable to primary human cells, including mutipotent and pluripotent cells, to uncover both the underlying mechanisms and phenotypic consequences of targeted translocations and other genomic rearrangements. Expand
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