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Promoterless gene targeting without nucleases ameliorates haemophilia B in mice

Nature (London), 2015-01, Vol.517 (7534), p.360-364

2015

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Zinc-finger nuclease-mediated gene correction using single AAV vector transduction and enhancement by Food and Drug Administration-approved drugs

Gene therapy, 2013-01, Vol.20 (1), p.35-42

2013

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Diamond Blackfan anemia is mediated by hyperactive Nemo-like kinase

Nature communications, 2020-07, Vol.11 (1), p.3344-3344, Article 3344

2020

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Interferon priming is essential for human CD34+ cell-derived plasmacytoid dendritic cell maturation and function

Nature communications, 2018-08, Vol.9 (1), p.3525-14, Article 3525

2018

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Chimeric Nucleases Stimulate Gene Targeting in Human Cells

Science (American Association for the Advancement of Science), 2003-05, Vol.300 (5620), p.763-763

2003

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CRISPR-based gene editing enables FOXP3 gene repair in IPEX patient cells

Science advances, 2020-05, Vol.6 (19), p.eaaz0571-eaaz0571

2020

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Self-complementary AAV mediates gene targeting and enhances endonuclease delivery for double-strand break repair

Gene therapy, 2010-09, Vol.17 (9), p.1175-1180

2010

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Treatment decision-making in sickle cell disease patients

Journal of community genetics, 2022-02, Vol.13 (1), p.143-151

2022

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Correction to: Treatment decision‑making in sickle cell disease patients

Journal of community genetics, 2022-02, Vol.13 (1), p.153-154

2022

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Efficient Gene Targeting Mediated by Adeno-Associated Virus and DNA Double-Strand Breaks

Molecular and Cellular Biology, 2003-05, Vol.23 (10), p.3558-3565

2003

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Spatially restricted expression of Dlx-1, Dlx-2 (Tes-1), Gbx-2, and Wnt- 3 in the embryonic day 12.5 mouse forebrain defines potential transverse and longitudinal segmental boundaries

The Journal of neuroscience, 1993-07, Vol.13 (7), p.3155-3172

1993

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Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements

Molecular therapy, 2019-01, Vol.27 (1), p.137-150

2019

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Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus

Blood, 2018-04, Vol.131 (17), p.1960-1973

2018

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MOF and Histone H4 Acetylation at Lysine 16 Are Critical for DNA Damage Response and Double-Strand Break Repair

Molecular and Cellular Biology, 2010-07, Vol.30 (14), p.3582-3595

2010

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Characteristics and Outcome of Children With Beckwith-Wiedemann Syndrome and Wilms’ Tumor: A Report From the National Wilms Tumor Study Group

Journal of clinical oncology, 2000-05, Vol.18 (10), p.2026-2031

2000

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Determining age and growth of abalone using stable oxygen isotopes: a tool for fisheries management

Fisheries research, 2005-05, Vol.72 (2), p.353-360

2005

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Gene targeting using zinc finger nucleases

Nature biotechnology, 2005-08, Vol.23 (8), p.967-973

2005

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DLX-2, MASH-1, and MAP-2 expression and bromodeoxyuridine incorporation define molecularly distinct cell populations in the embryonic mouse forebrain

The Journal of neuroscience, 1994-11, Vol.14 (11), p.6370-6383

1994

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Mammalian Gene Targeting with Designed Zinc Finger Nucleases

Molecular therapy, 2006-02, Vol.13 (2), p.438-446

Porteus, Matthew H

2006

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Creating higher titer lentivirus with caffeine

Human gene therapy, 2011-01, Vol.22 (1), p.93-100

2011

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Isolation and characterization of a novel cDNA clone encoding a homeodomain that is developmentally regulated in the ventral forebrain

Neuron (Cambridge, Mass.), 1991-08, Vol.7 (2), p.221-229

1991

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Highly efficient endogenous human gene correction using designed zinc-finger nucleases

Nature, 2005-06, Vol.435 (7042), p.646-651

2005

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Gene editing using CRISPR enables FOXP3 gene repair in HSPCs and IPEX patient T cells

Cytotherapy (Oxford, England), 2020-05, Vol.22 (5), p.S20-S20

2020

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Sequence, organization, and transcription of the Dlx-1 and Dlx-2 locus

Genomics (San Diego, Calif.), 1996-08, Vol.35 (3), p.473-485

1996

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Zinc finger nucleases: custom-designed molecular scissors for genome engineering of plant and mammalian cells

Nucleic acids research, 2005-01, Vol.33 (18), p.5978-5990

2005

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Promoterless gene targeting without nucleases ameliorates haemophilia B in mice

Zinc-finger nuclease-mediated gene correction using single AAV vector transduction and enhancement by Food and Drug Administration-approved drugs

Diamond Blackfan anemia is mediated by hyperactive Nemo-like kinase

Interferon priming is essential for human CD34+ cell-derived plasmacytoid dendritic cell maturation and function

Chimeric Nucleases Stimulate Gene Targeting in Human Cells

CRISPR-based gene editing enables FOXP3 gene repair in IPEX patient cells

Self-complementary AAV mediates gene targeting and enhances endonuclease delivery for double-strand break repair

Treatment decision-making in sickle cell disease patients

Correction to: Treatment decision‑making in sickle cell disease patients

Efficient Gene Targeting Mediated by Adeno-Associated Virus and DNA Double-Strand Breaks

Spatially restricted expression of Dlx-1, Dlx-2 (Tes-1), Gbx-2, and Wnt- 3 in the embryonic day 12.5 mouse forebrain defines potential transverse and longitudinal segmental boundaries

Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements

Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus

MOF and Histone H4 Acetylation at Lysine 16 Are Critical for DNA Damage Response and Double-Strand Break Repair

Characteristics and Outcome of Children With Beckwith-Wiedemann Syndrome and Wilms’ Tumor: A Report From the National Wilms Tumor Study Group

Determining age and growth of abalone using stable oxygen isotopes: a tool for fisheries management

Gene targeting using zinc finger nucleases

DLX-2, MASH-1, and MAP-2 expression and bromodeoxyuridine incorporation define molecularly distinct cell populations in the embryonic mouse forebrain

Mammalian Gene Targeting with Designed Zinc Finger Nucleases

Creating higher titer lentivirus with caffeine

Isolation and characterization of a novel cDNA clone encoding a homeodomain that is developmentally regulated in the ventral forebrain

Highly efficient endogenous human gene correction using designed zinc-finger nucleases

Gene editing using CRISPR enables FOXP3 gene repair in HSPCs and IPEX patient T cells

Sequence, organization, and transcription of the Dlx-1 and Dlx-2 locus

Zinc finger nucleases: custom-designed molecular scissors for genome engineering of plant and mammalian cells

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