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Journal Articles

the NADPH Oxidase Catalytic Subunit Nox2 Displays Differential Roles in Arterial Vs. Venous Thrombosis

Vijay Sonkar, PhD, Melissa J Jensen, BS, Steven R. Lentz, MD PhD, Sanjana Dayal, PhD

Blood (2016) 128 (22): 4907.

https://doi.org/10.1182/blood.V128.22.4907.4907

Published: 2016

Journal Articles

High Efficiency Gene Correction in Hematopoietic Cells By Template-Free Crispr/Cas9 Genome Editing

Frank Schnütgen, PhD, Duran Sürün, Joachim Schwäble, PhD, Ana Tomasovic, PhD, Ralf Kühn, PhD, Stefan Stein, PhD, Nina Kurrle, PhD, Hubert Serve, MD, Erhard Seifried, MD PhD, Harald von Melchner, MD PhD

Blood (2016) 128 (22): 3507.

https://doi.org/10.1182/blood.V128.22.3507.3507

Published: 2016

Journal Articles

High Titer Lentivector from a Stable Lenti-Producer Efficiently Corrects CD34+ Hematopoietic Stem Cells from Patients with p47phox-Deficient Chronic Granulomatous Disease

Uimook Choi, PhD, Narda Theobald, Throm E Robert, PhD, John Gray, PhD, David J. Rawlings, MD, Brian P. Sorrentino, MD, Harry Malech, Suk See De Ravin, MD

Blood (2015) 126 (23): 2036.

https://doi.org/10.1182/blood.V126.23.2036.2036

Published: 2015

Journal Articles

Deletion of OLFM4 Rescues Defective Host Defense Against Staphylococcus Aureus , but Not Aspergillus Fumigatus in Murine X-Linked Chronic Granulomatous Disease

Wenli Liu, MD, Janyce A Sugui, Hongzhen Li, Kyung J Kwon-Chung, Griffin P. Rodgers, MD

Blood (2012) 120 (21): 3276.

https://doi.org/10.1182/blood.V120.21.3276.3276

Published: 2012

Journal Articles

Integration-Mediated Activation of PRDM16 and HMGA2 in Multiple Clones without Adverse Hematopoietic Consequences Following Transplant of Autologous MGMTP140K Gene-Modified CD34 + Cells

Jennifer E Adair, PhD, Brian C Beard, PhD, Grant Trobridge, PhD, Frederic Bushman, PhD, Maciej M Mrugala, MD PhD, Hans-Peter Kiem, MD

Blood (2011) 118 (21): 2053.

https://doi.org/10.1182/blood.V118.21.2053.2053

Published: 2011

Journal Articles

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

Jizhong Zou, Colin L. Sweeney, Bin-Kuan Chou, Uimook Choi, Jason Pan, Hongmei Wang, Sarah N. Dowey, Linzhao Cheng, Harry L. Malech

Blood (2011) 117 (21): 5561–5572.

https://doi.org/10.1182/blood-2010-12-328161

Published: 2011

Journal Articles

Correcting CGD safely, iPSo facto

Luigi D. Notarangelo

Blood (2011) 117 (21): 5554–5556.

https://doi.org/10.1182/blood-2011-04-344119

Published: 2011

Journal Articles

Antibody targeting KIT as pretransplantation conditioning in immunocompetent mice

Xingkui Xue, Nancy K. Pech, W. Christopher Shelley, Edward F. Srour, Mervin C. Yoder, Mary C. Dinauer

Blood (2010) 116 (24): 5419–5422.

https://doi.org/10.1182/blood-2010-07-295949

Published: 2010

Includes: Supplemental data

Journal Articles

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

Elizabeth M. Kang, Uimook Choi, Narda Theobald, Gilda Linton, Debra A. Long Priel, Doug Kuhns, Harry L. Malech

Blood (2010) 115 (4): 783–791.

https://doi.org/10.1182/blood-2009-05-222760

Published: 2010

Includes: Supplemental data

Journal Articles

Retroviral Gene Therapy for X-Linked Chronic Granulomatous Disease: Results from Phase I/II Trial.

Joong Gon Kim, MD, PhD, Hyo Seop Ahn, MD, PhD, Hyoung Jin Kang, MD, PhD, Sujeong Kim, PhD, Youngtae Hong, PhD, Chang-Wan Joo, MS, Nam-Kyung Yoon, MS, Sunyoung Kim, DPhil

Blood (2008) 112 (11): 2349.

https://doi.org/10.1182/blood.V112.11.2349.2349

Published: 2008

Journal Articles

Treatment of Infections in Patients with X-Linked Chronic Granulomatous Disease (XCGD) with Gene Therapy.

Elizabeth M. Kang, MD, Gilda Linton, Narda Theobald, Sandra Anaya O’Brien, RN, Dianne Hilligoss, CNP, Harry L. Malech, MD

Blood (2007) 110 (11): 504.

https://doi.org/10.1182/blood.V110.11.504.504

Published: 2007

Journal Articles

Correction of Human X-Linked Chronic Granulomatous Disease (X-CGD) Following Transduction of X-CGD CD34+ Cells with a Modified RD114-Pseudotyped Simian Immunodeficiency Virus Lentiviral Vector in a NOD/SCID Mouse Xenograft Model.

Nora Naumann, MD, PhD, Suk See De Ravin, MD, PhD, Uimook Choi, PhD, Morvarid Moayeri, MD, PhD, Yasuhiro Ikeda, DVM, PhD, Harry L. Malech, MD

Blood (2006) 108 (11): 3278.

https://doi.org/10.1182/blood.V108.11.3278.3278

Published: 2006

Journal Articles

Biased Engraftment of Retrovirus-Transduced Marrow in Murine X-CGD Following Submyeloablative Conditioning.

Mohammed Sadat, Sara Dirscherl, Nancy Pech, Robert Getty, Scott Cross, Cecilia Barese, W. Scott Goebel, Mervin C. Yoder, Sean Mooney, Manuel Grez, Kenneth Cornetta, Mary C. Dinauer

Blood (2006) 108 (11): 3285.

https://doi.org/10.1182/blood.V108.11.3285.3285

Published: 2006

Journal Articles

Long-Term Clinical Follow-Up and Safety/Toxicity Analysis in 3 X-CGD Patients Treated by Gene Therapy and Non-Myeloablative Conditioning.

Marion G. Ott, MD, Manfred Schmidt, PhD, Stefan Stein, PhD, Kerstin Schwarzwaelder, Ulrich Siler, PhD, Ulrike Koehl, PhD, Klaus Kuehlcke, PhD, Andrea Schilz, PhD, Christoph v. Kalle, PhD, Dieter Hoelzer, MD, Reinhard Seger, MD, Manuel Grez, PhD

Blood (2006) 108 (11): 199.

https://doi.org/10.1182/blood.V108.11.199.199

Published: 2006

Journal Articles

Reconstitution of Neutrophil NADPH Oxidase Activity in Murine X-CGD Following Transplantation of Retrovirus-Transduced Marrow: Potential Impact of Submyeloablative Conditioning.

Mohammed Sadat, MBBS, PhD, Cecilia Barese, MD, Nancy Pech, W. Scott Goebel, MD, PhD, Mervin C. Yoder, MD, Sean Mooney, PhD, Kenneth Cornetta, MD, Manuel Grez, PhD, Mary C. Dinauer, MD, PhD

Blood (2005) 106 (11): 3050.

https://doi.org/10.1182/blood.V106.11.3050.3050

Published: 2005

Journal Articles

Long-Term Follow-Up of Patients Treated by Gene Therapy for X-Linked Chronic Granulomatous Disease.

Marion G. Ott, MD, Manuel Grez, PhD, Stefan Stein, PhD, Ulrich Siler, PhD, Ulrike Koehl, PhD, Klaus Kuehlcke, PhD, Manfred Schmidt, MD, Andrea Schilz, PhD, Hana Kunkel, Sonja Naundorf, PhD, Andrea Brinkmann, Moustapha Hassan, PhD, Christof von Kalle, MD, Adrian Trasher, MD, Reinhard Seger, MD, Dieter Hoelzer, MD

Blood (2005) 106 (11): 194.

https://doi.org/10.1182/blood.V106.11.194.194

Published: 2005

Journal Articles

Unusual late presentation of X-linked chronic granulomatous disease in an adult female with a somatic mosaic for a novel mutation in CYBB

Baruch Wolach, Yitshak Scharf, Ronit Gavrieli, Martin de Boer, Dirk Roos

Blood (2005) 105 (1): 61–66.

https://doi.org/10.1182/blood-2004-02-0675

Published: 2005

Journal Articles

Granulocyte Colony-Stimulating Factor Prior to Low-Dose Total Body Irradiation as Conditioning Regimen in Hematopoietic Stem Cell Transplantation for Gene Therapy.

Cecilia N. Barese, MD, W. Scott Goebel, MD, PhD, Nancy K. Pech, Justin Meyers, Mary C. Dinauer, MD, PhD

Blood (2004) 104 (11): 1193.

https://doi.org/10.1182/blood.V104.11.1193.1193

Published: 2004

Journal Articles

Gene Therapy for X-Linked Chronic Granulomatous Disease.

Marion G. Ott, MD, Stefan Stein, PhD, Ulrike Koehl, PhD, Andrea Schilz, PhD, Klaus Kuhlcke, PhD, Manfred Schmidt, PhD, Christof von Kalle, MD, Moustapha Hassan, MD, Ulrich Siler, PhD, Dieter Hoelzer, MD, Reinhard Seger, MD, Manuel Grez, PhD

Blood (2004) 104 (11): 409.

https://doi.org/10.1182/blood.V104.11.409.409

Published: 2004

Journal Articles

Third-generation, self-inactivating gp91 phox lentivector corrects the oxidase defect in NOD/SCID mouse–repopulating peripheral blood–mobilized CD34 + cells from patients with X-linked chronic granulomatous disease

Joachim Roesler, Sebastian Brenner, Anatoly A. Bukovsky, Narda Whiting-Theobald, Thomas Dull, Michael Kelly, Curt I. Civin, Harry L. Malech

Blood (2002) 100 (13): 4381–4390.

https://doi.org/10.1182/blood-2001-12-0165

Published: 2002

NARROW

the NADPH Oxidase Catalytic Subunit Nox2 Displays Differential Roles in Arterial Vs. Venous Thrombosis

High Efficiency Gene Correction in Hematopoietic Cells By Template-Free Crispr/Cas9 Genome Editing

High Titer Lentivector from a Stable Lenti-Producer Efficiently Corrects CD34+ Hematopoietic Stem Cells from Patients with p47phox-Deficient Chronic Granulomatous Disease

Deletion of OLFM4 Rescues Defective Host Defense Against Staphylococcus Aureus , but Not Aspergillus Fumigatus in Murine X-Linked Chronic Granulomatous Disease

Integration-Mediated Activation of PRDM16 and HMGA2 in Multiple Clones without Adverse Hematopoietic Consequences Following Transplant of Autologous MGMTP140K Gene-Modified CD34 + Cells

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

Correcting CGD safely, iPSo facto

Antibody targeting KIT as pretransplantation conditioning in immunocompetent mice

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

Retroviral Gene Therapy for X-Linked Chronic Granulomatous Disease: Results from Phase I/II Trial.

Treatment of Infections in Patients with X-Linked Chronic Granulomatous Disease (XCGD) with Gene Therapy.

Correction of Human X-Linked Chronic Granulomatous Disease (X-CGD) Following Transduction of X-CGD CD34+ Cells with a Modified RD114-Pseudotyped Simian Immunodeficiency Virus Lentiviral Vector in a NOD/SCID Mouse Xenograft Model.

Biased Engraftment of Retrovirus-Transduced Marrow in Murine X-CGD Following Submyeloablative Conditioning.

Long-Term Clinical Follow-Up and Safety/Toxicity Analysis in 3 X-CGD Patients Treated by Gene Therapy and Non-Myeloablative Conditioning.

Reconstitution of Neutrophil NADPH Oxidase Activity in Murine X-CGD Following Transplantation of Retrovirus-Transduced Marrow: Potential Impact of Submyeloablative Conditioning.

Long-Term Follow-Up of Patients Treated by Gene Therapy for X-Linked Chronic Granulomatous Disease.

Unusual late presentation of X-linked chronic granulomatous disease in an adult female with a somatic mosaic for a novel mutation in CYBB

Granulocyte Colony-Stimulating Factor Prior to Low-Dose Total Body Irradiation as Conditioning Regimen in Hematopoietic Stem Cell Transplantation for Gene Therapy.

Gene Therapy for X-Linked Chronic Granulomatous Disease.

Third-generation, self-inactivating gp91 phox lentivector corrects the oxidase defect in NOD/SCID mouse–repopulating peripheral blood–mobilized CD34 + cells from patients with X-linked chronic granulomatous disease

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American Society of Hematology

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ASH Publications

American Society of Hematology

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