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Neutrophil extracellular traps enriched in oxidized mitochondrial DNA are interferogenic and contribute to lupus-like disease

Nature medicine, 2016-02, Vol.22 (2), p.146-153

2016

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Differentiation of human and murine induced pluripotent stem cells to microglia-like cells

Nature neuroscience, 2017-05, Vol.20 (5), p.753

2017

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Pyocyanin-enhanced neutrophil extracellular trap formation requires the NADPH oxidase

PloS one, 2013-01, Vol.8 (1), p.e54205-e54205

2013

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Oxidase-deficient neutrophils from X-linked chronic granulomatous disease iPS cells: functional correction by zinc finger nuclease–mediated safe harbor targeting

Blood, 2011-05, Vol.117 (21), p.5561-5572

2011

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Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency

Science translational medicine, 2016-04, Vol.8 (335), p.335ra57

2016

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Lentiviral Gene Therapy for Artemis-Deficient SCID

The New England journal of medicine, 2022-12, Vol.387 (25), p.2344-2355

2022

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Enhancers are major targets for murine leukemia virus vector integration

Journal of virology, 2014-04, Vol.88 (8), p.4504-4513

2014

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Retrovirus gene therapy for X-linked chronic granulomatous disease can achieve stable long-term correction of oxidase activity in peripheral blood neutrophils

Blood, 2010-01, Vol.115 (4), p.783-791

2010

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Lentivector cryptic splicing mediates increase in CD34+ clones expressing truncated HMGA2 in human X-linked severe combined immunodeficiency

Nature communications, 2022-06, Vol.13 (1), p.3710-3710, Article 3710

2022

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Chronic granulomatous disease as a risk factor for autoimmune disease

Journal of allergy and clinical immunology, 2008-12, Vol.122 (6), p.1097-1103

2008

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Treatment by CRISPR-Cas9 Gene Editing — A Proof of Principle

The New England journal of medicine, 2021-01, Vol.384 (3), p.286-287

Malech, Harry L

2021

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A phase III randomized crossover trial of plerixafor versus G-CSF for treatment of WHIM syndrome

The Journal of clinical investigation, 2023-10, Vol.133 (19), p.1-16

2023

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Transgene-free iPSCs generated from small volume peripheral blood nonmobilized CD34+ cells

Blood, 2013-04, Vol.121 (14), p.e98-e107

2013

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Molecular Analysis of Neutrophil Differentiation from Human Induced Pluripotent Stem Cells Delineates the Kinetics of Key Regulators of Hematopoiesis

Stem cells (Dayton, Ohio), 2016-06, Vol.34 (6), p.1513-1526

2016

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Granulocyte transfusions in patients with chronic granulomatous disease and refractory infections: The NIH experience

Journal of allergy and clinical immunology, 2017-08, Vol.140 (2), p.622-625

2017

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Lentivirus Mediated Correction of Artemis-Deficient Severe Combined Immunodeficiency

Human gene therapy, 2017-01, Vol.28 (1), p.112

2017

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Hematologically important mutations: X-linked chronic granulomatous disease (third update)

Blood cells, molecules, & diseases, 2010-10, Vol.45 (3), p.246-265

2010

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The Changing Paradigm of Management of Liver Abscesses in Chronic Granulomatous Disease

Clinical infectious diseases, 2018-04, Vol.66 (9), p.1427-1434

2018

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Aspergillus nidulans Infection in Chronic Granulomatous Disease

Medicine (Baltimore), 1998-09, Vol.77 (5), p.345-354

1998

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Hypomorphic Rag mutations can cause destructive midline granulomatous disease

Blood, 2010-08, Vol.116 (8), p.1263-1271

2010

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Gastrointestinal involvement in chronic granulomatous disease

Pediatrics (Evanston), 2004-08, Vol.114 (2), p.462

2004

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Efficient construction of producer cell lines for a SIN lentiviral vector for SCID-X1 gene therapy by concatemeric array transfection

Blood, 2009-05, Vol.113 (21), p.5104-5110

2009

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CRISPR-Cas9-AAV versus lentivector transduction for genome modification of X-linked severe combined immunodeficiency hematopoietic stem cells

Frontiers in immunology, 2023-01, Vol.13, p.1067417-1067417

2023

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Granulibacter bethesdensis , a Pathogen from Patients with Chronic Granulomatous Disease, Produces a Penta-Acylated Hypostimulatory Glycero-D-talo-oct-2-ulosonic Acid-Lipid A Glycolipid (Ko-Lipid A)

International journal of molecular sciences, 2021-03, Vol.22 (7), p.3303

2021

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Hematologically important mutations: The autosomal recessive forms of chronic granulomatous disease (second update)

Blood cells, molecules, & diseases, 2010-04, Vol.44 (4), p.291-299

2010

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Neutrophil extracellular traps enriched in oxidized mitochondrial DNA are interferogenic and contribute to lupus-like disease

Differentiation of human and murine induced pluripotent stem cells to microglia-like cells

Pyocyanin-enhanced neutrophil extracellular trap formation requires the NADPH oxidase

Oxidase-deficient neutrophils from X-linked chronic granulomatous disease iPS cells: functional correction by zinc finger nuclease–mediated safe harbor targeting

Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency

Lentiviral Gene Therapy for Artemis-Deficient SCID

Enhancers are major targets for murine leukemia virus vector integration

Retrovirus gene therapy for X-linked chronic granulomatous disease can achieve stable long-term correction of oxidase activity in peripheral blood neutrophils

Lentivector cryptic splicing mediates increase in CD34+ clones expressing truncated HMGA2 in human X-linked severe combined immunodeficiency

Chronic granulomatous disease as a risk factor for autoimmune disease

Treatment by CRISPR-Cas9 Gene Editing — A Proof of Principle

A phase III randomized crossover trial of plerixafor versus G-CSF for treatment of WHIM syndrome

Transgene-free iPSCs generated from small volume peripheral blood nonmobilized CD34+ cells

Molecular Analysis of Neutrophil Differentiation from Human Induced Pluripotent Stem Cells Delineates the Kinetics of Key Regulators of Hematopoiesis

Granulocyte transfusions in patients with chronic granulomatous disease and refractory infections: The NIH experience

Lentivirus Mediated Correction of Artemis-Deficient Severe Combined Immunodeficiency

Hematologically important mutations: X-linked chronic granulomatous disease (third update)

The Changing Paradigm of Management of Liver Abscesses in Chronic Granulomatous Disease

Aspergillus nidulans Infection in Chronic Granulomatous Disease

Hypomorphic Rag mutations can cause destructive midline granulomatous disease

Gastrointestinal involvement in chronic granulomatous disease

Efficient construction of producer cell lines for a SIN lentiviral vector for SCID-X1 gene therapy by concatemeric array transfection

CRISPR-Cas9-AAV versus lentivector transduction for genome modification of X-linked severe combined immunodeficiency hematopoietic stem cells

Granulibacter bethesdensis , a Pathogen from Patients with Chronic Granulomatous Disease, Produces a Penta-Acylated Hypostimulatory Glycero-D-talo-oct-2-ulosonic Acid-Lipid A Glycolipid (Ko-Lipid A)

Hematologically important mutations: The autosomal recessive forms of chronic granulomatous disease (second update)

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