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Aberrant chromosome morphology in human cells defective for Holliday junction resolution

Nature (London), 2011-03, Vol.471 (7340), p.642-646

2011

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In vivo genome editing of the albumin locus as a platform for protein replacement therapy

Blood, 2015-10, Vol.126 (15), p.1777-1784

2015

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GlcNAc-Asn is a biomarker for NGLY1 deficiency

Journal of biochemistry (Tokyo), 2022-02, Vol.171 (2), p.177-186

2022

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AAV9-NGLY1 gene replacement therapy improves phenotypic and biomarker endpoints in a rat model of NGLY1 Deficiency

Molecular therapy. Methods & clinical development, 2022-12, Vol.27, p.259-271

2022

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AAV2/6 Gene Therapy in a Murine Model of Fabry Disease Results in Supraphysiological Enzyme Activity and Effective Substrate Reduction

Molecular therapy. Methods & clinical development, 2020-09, Vol.18, p.607-619

2020

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RIDDLE immunodeficiency syndrome is linked to defects in 53BP1-mediated DNA damage signaling

Proceedings of the National Academy of Sciences - PNAS, 2007-10, Vol.104 (43), p.16910-16915

2007

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ZFN-Mediated Gene Targeting at the Albumin Locus in Liver Results in Therapeutic Levels of Human FIX in Mice and Non-Human Primates

Blood, 2015-12, Vol.126 (23), p.200-200

2015

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DNA-PKcs Function Regulated Specifically by Protein Phosphatase 5

Proceedings of the National Academy of Sciences - PNAS, 2004-02, Vol.101 (5), p.1247-1252

2004

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Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing

Molecular therapy, 2018-04, Vol.26 (4), p.1127-1136

2018

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ZFN-mediated in vivo gene editing in hepatocytes leads to supraphysiologic α-Gal A activity and effective substrate reduction in Fabry mice

Molecular therapy, 2021-11, Vol.29 (11), p.3230-3242

2021

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ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome

Molecular therapy, 2019-01, Vol.27 (1), p.178-187

2019

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Liver-targeted AAV gene therapy vectors produced by a clinical scale manufacturing process result in high, continuous therapeutic levels of enzyme activity and effective substrate reduction in mouse model of Fabry disease

Molecular genetics and metabolism, 2019-02, Vol.126 (2), p.S77-S77

2019

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ZFN-mediated in vivo genome editing results in therapeutic levels of α-galactosidase A and effective substrate reduction in Fabry knockout mice

Molecular genetics and metabolism, 2018-02, Vol.123 (2), p.S113-S113

2018

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Liver-based expression of the human alpha-galactosidase A gene in a murine Fabry model results in continuous therapeutic levels of enzyme activity and effective substrate reduction

Molecular genetics and metabolism, 2018-02, Vol.123 (2), p.S68-S68

2018

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In Vivo Genome Editing in Neonatal Mouse Liver Preferentially Utilizes Homology Directed Repair

Blood, 2015-12, Vol.126 (23), p.4422-4422

2015

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Liver-based expression of the human alpha-galactosidase A gene (GLA) in a murine Fabry model results in continuous supra-physiological enzyme activity and effective substrate reduction

Molecular genetics and metabolism, 2017-01, Vol.120 (1-2), p.S69-S69

2017

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Update on phase 1/2 clinical trials for MPS I and MPS II using ZFN-mediated in vivo genome editing

Molecular genetics and metabolism, 2018-02, Vol.123 (2), p.S59-S60

2018

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ZFN-mediated in vivo genome editing of hepatocytes results in phenotypic correction in murine MPS I and MPS II models

Molecular genetics and metabolism, 2018-02, Vol.123 (2), p.S146-S147

2018

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484. In Vivo Zinc-Finger Nuclease Mediated Iduronate-2-Sulfatase (IDS) Target Gene Insertion and Correction of Metabolic Disease in a Mouse Model of Mucopolysaccharidosis Type II (MPS II)

Molecular therapy, 2016-05, Vol.24, p.S192-S192

2016

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485. ZFN-Mediated Liver-Targeting Gene Therapy Corrects Systemic and Neurological Disease of Mucopolysaccharidosis Type I

Molecular therapy, 2016-05, Vol.24, p.S192-S193

2016

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ZFN-mediated genome editing of albumin “safe harbor” in vivo results in supraphysiological levels of human IDS, IDUA and GBA in mice

Molecular genetics and metabolism, 2015-02, Vol.114 (2), p.S36-S36

2015

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ZFN-mediated in vivo genome editing results in phenotypic correction in murine MPS I and MPS II models

Molecular genetics and metabolism, 2017-01, Vol.120 (1-2), p.S41-S41

2017

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479. ZFN-Mediated In Vivo Genome Editing Results in Supraphysiological Levels of Lysosomal Enzymes Deficient in Hunter and Hurler Syndrome and Gaucher Disease

Molecular therapy, 2015-05, Vol.23, p.S190-S190

2015

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ZFN-mediated in vivo genome editing results in supraphysiological levels of human iduronate 2-sulfatase and phenotypic correction in a murine MPS II model

Molecular genetics and metabolism, 2016-02, Vol.117 (2), p.S40-S40

2016

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ZFN-mediated correction of murine MPS I model by expression of the human IDUA cDNA from the albumin “safe harbor” locus

Molecular genetics and metabolism, 2016-02, Vol.117 (2), p.S89-S89

2016

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Aberrant chromosome morphology in human cells defective for Holliday junction resolution

In vivo genome editing of the albumin locus as a platform for protein replacement therapy

GlcNAc-Asn is a biomarker for NGLY1 deficiency

AAV9-NGLY1 gene replacement therapy improves phenotypic and biomarker endpoints in a rat model of NGLY1 Deficiency

AAV2/6 Gene Therapy in a Murine Model of Fabry Disease Results in Supraphysiological Enzyme Activity and Effective Substrate Reduction

RIDDLE immunodeficiency syndrome is linked to defects in 53BP1-mediated DNA damage signaling

ZFN-Mediated Gene Targeting at the Albumin Locus in Liver Results in Therapeutic Levels of Human FIX in Mice and Non-Human Primates

DNA-PKcs Function Regulated Specifically by Protein Phosphatase 5

Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing

ZFN-mediated in vivo gene editing in hepatocytes leads to supraphysiologic α-Gal A activity and effective substrate reduction in Fabry mice

ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome

Liver-targeted AAV gene therapy vectors produced by a clinical scale manufacturing process result in high, continuous therapeutic levels of enzyme activity and effective substrate reduction in mouse model of Fabry disease

ZFN-mediated in vivo genome editing results in therapeutic levels of α-galactosidase A and effective substrate reduction in Fabry knockout mice

Liver-based expression of the human alpha-galactosidase A gene in a murine Fabry model results in continuous therapeutic levels of enzyme activity and effective substrate reduction

In Vivo Genome Editing in Neonatal Mouse Liver Preferentially Utilizes Homology Directed Repair

Liver-based expression of the human alpha-galactosidase A gene (GLA) in a murine Fabry model results in continuous supra-physiological enzyme activity and effective substrate reduction

Update on phase 1/2 clinical trials for MPS I and MPS II using ZFN-mediated in vivo genome editing

ZFN-mediated in vivo genome editing of hepatocytes results in phenotypic correction in murine MPS I and MPS II models

484. In Vivo Zinc-Finger Nuclease Mediated Iduronate-2-Sulfatase (IDS) Target Gene Insertion and Correction of Metabolic Disease in a Mouse Model of Mucopolysaccharidosis Type II (MPS II)

485. ZFN-Mediated Liver-Targeting Gene Therapy Corrects Systemic and Neurological Disease of Mucopolysaccharidosis Type I

ZFN-mediated genome editing of albumin “safe harbor” in vivo results in supraphysiological levels of human IDS, IDUA and GBA in mice

ZFN-mediated in vivo genome editing results in phenotypic correction in murine MPS I and MPS II models

479. ZFN-Mediated In Vivo Genome Editing Results in Supraphysiological Levels of Lysosomal Enzymes Deficient in Hunter and Hurler Syndrome and Gaucher Disease

ZFN-mediated in vivo genome editing results in supraphysiological levels of human iduronate 2-sulfatase and phenotypic correction in a murine MPS II model

ZFN-mediated correction of murine MPS I model by expression of the human IDUA cDNA from the albumin “safe harbor” locus

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