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Journal Articles

BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression

Barbara A. Konkle, Christopher E. Walsh, Miguel A. Escobar, Neil C. Josephson, Guy Young, Annette von Drygalski, Scott W. J. McPhee, R. Jude Samulski, Ivan Bilic, Maurus de la Rosa, Birgit M. Reipert, Hanspeter Rottensteiner, Friedrich Scheiflinger, John C. Chapin, Bruce Ewenstein, Paul E. Monahan

Blood (2021) 137 (6): 763–774.

https://doi.org/10.1182/blood.2019004625

Published: 2021

Includes: Supplemental data

Journal Articles

Results of Genetic Analysis of 11,341 Participants Enrolled in the My Life, Our Future (MLOF) Hemophilia Genotyping Initiative

Jill M Johnsen, MD, Shelley N Fletcher, Angela Dove, Haley McCracken, Beth K Martin, Martin Kircher, Neil C Josephson, MD, Jay Shendure, Sarah Ruuska, MPH, Leonard A. Valentino, MD, Glenn Pierce, MD PhD, Crystal Watson, BS, Dunlei Cheng, PhD, Michael Recht, MD PhD, Barbara A. Konkle, MD

Blood (2020) 136 (Supplement 1): 19.

https://doi.org/10.1182/blood-2020-140649

Published: 2020

Journal Articles

Real-World Clinical and Economic Burden of Hematopoietic Cell Transplantation Among a Large US Commercially Insured Populationhematopoietic CELL Transplantation Among a Large US Commercially Insured Population

Neil C Josephson, MD, Phillip M. Garfin, MDPhD, Akshara Richhariya, PhD, Machaon Bonafede, McMorrow Donna, Qian Cai, Miguel Perales, MD

Blood (2016) 128 (22): 2368.

https://doi.org/10.1182/blood.V128.22.2368.2368

Published: 2016

Journal Articles

Toxicity Burden of Bleomycin Treatment in Hodgkin Lymphoma: A Systematic Literature Review

Kathleen M Fox, PhD, Neil C Josephson, MD, Akshara Richhariya, PhD

Blood (2016) 128 (22): 3566.

https://doi.org/10.1182/blood.V128.22.3566.3566

Published: 2016

Journal Articles

Novel Approach to and Results of Genetic Analysis of 3000 Hemophilia Patients Enrolled in the MyLifeOurFuture Initiative

Jill Johnsen, Shelley N Fletcher, Haley Huston, Sarah Roberge, Beth K Martin, Martin Kircher, Neil C Josephson, Jay Shendure, Sarah Ruuska, Marion A Koerper, Leslie Meltzer, Glenn F Pierce, Diane Aschman, Pharm D, Barbara Konkle, MD

Blood (2016) 128 (22): 205.

https://doi.org/10.1182/blood.V128.22.205.205

Published: 2016

Journal Articles

Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A

Blood (2014) 123 (3): 317–325.

https://doi.org/10.1182/blood-2013-10-529974

Published: 2014

Includes: Supplemental data

Journal Articles

Phosphorylation Of The Cytoplasmic Tyrosine Residues Of c-Mpl Is Not Required To Preserve Intact Receptor Dependent Hematopoietic Stem Cell Survival Signaling

Jacqueline Roy, PhD, Angela Epp, Junli Feng, Sandra Ziegler, Radek C. Skoda, M.D., Neil C. Josephson, MD

Blood (2013) 122 (21): 3679.

https://doi.org/10.1182/blood.V122.21.3679.3679

Published: 2013

Journal Articles

Safety and prolonged activity of recombinant factor VIII Fc fusion protein in hemophilia A patients

Jerry S. Powell, Neil C. Josephson, Doris Quon, Margaret V. Ragni, Gregory Cheng, Ella Li, Haiyan Jiang, Lian Li, Jennifer A. Dumont, Jaya Goyal, Xin Zhang, Jurg Sommer, Justin McCue, Margaret Barbetti, Alvin Luk, Glenn F. Pierce

Blood (2012) 119 (13): 3031–3037.

https://doi.org/10.1182/blood-2011-09-382846

Published: 2012

Includes: Supplemental data

Journal Articles

Recombinant factor IX-Fc fusion protein (rFIXFc) demonstrates safety and prolonged activity in a phase 1/2a study in hemophilia B patients

Blood (2012) 119 (3): 666–672.

https://doi.org/10.1182/blood-2011-07-367003

Published: 2012

Includes: Supplemental data

Journal Articles

Determination of Anti-Factor VIIII Antibody Titers, Isotypes and Epitopes by SPR Analysis of Human Plasma

Kenneth B Lewis, Ph.D., Phuong-Cac Nguyen, Ph.D., Jason T. Schuman, Ph.D., Christopher E. Walsh, MD, PhD, Neil C. Josephson, MD, Tom E. Howard, MD, PhD, Dana C. Matthews, MD, Nigel S. Key, MBChB, Kathleen P. Pratt, Ph.D.

Blood (2011) 118 (21): 202.

https://doi.org/10.1182/blood.V118.21.202.202

Published: 2011

Journal Articles

Factor VIII Mutation and Desmopressin-Responsiveness in 63 Patients with Mild Hemophilia A

Danielle Nance, MD, Shelley Nakaya Fletcher, Doug C Bolgiano, MS, Arthur R. Thompson, MD, PhD, Neil C. Josephson, MD, Barbara A. Konkle, M.D.

Blood (2011) 118 (21): 1203.

https://doi.org/10.1182/blood.V118.21.1203.1203

Published: 2011

Journal Articles

Survey of Current Prophylaxis Practices and Bleeding Characteristics of Children with Severe Hemophilia A In U.S. Hemophilia Treatment Centers.

Margaret V. Ragni, MD, MPH, Patrick F. Fogarty, MD, Neil C. Josephson, MD, Anne T. Neff, MD, Leslie Raffini, MD, Craig M. Kessler, MD

Blood (2010) 116 (21): 3653.

https://doi.org/10.1182/blood.V116.21.3653.3653

Published: 2010

Journal Articles

Incomplete restoration of Mpl expression in the mpl −/− mouse produces partial correction of the stem cell–repopulating defect and paradoxical thrombocytosis

Brian J. Lannutti, Angela Epp, Jacqueline Roy, Junmei Chen, Neil C. Josephson

Blood (2009) 113 (8): 1778–1785.

https://doi.org/10.1182/blood-2007-11-124859

Published: 2009

Includes: Supplemental data

Journal Articles

Induction of Tolerance to FVIII in Hemophilic Mice by Delivery of Apoptotic Syngeneic Fibroblasts Expressing a FVIII Transgene.

Rui-Jun Su, MD, PhD, Laura Stewart, Angela Epp, Steven W. Pipe, MD, Neil C. Josephson, MD

Blood (2006) 108 (11): 768.

https://doi.org/10.1182/blood.V108.11.768.768

Published: 2006

Journal Articles

Reduction of Anti-FVIII Inhibitor Titers in Hemophilic Mice Infused with Syngeneic Apoptotic Cells Expressing a Human FVIII Transgene.

Rui-Jun Su, MD, PhD, Laura Stewart, Steven W. Pipe, MD, Neil C. Josephson, MD

Blood (2005) 106 (11): 216.

https://doi.org/10.1182/blood.V106.11.216.216

Published: 2005

Journal Articles

Transduction of Primate Cells with Feline Foamy Virus Envelope Pseudotyped Prototype Foamy Virus Vectors.

RuiJun Su, Rency L. Rosales, Martin Lochelt, Neil C. Josephson

Blood (2004) 104 (11): 5276.

https://doi.org/10.1182/blood.V104.11.5276.5276

Published: 2004

Journal Articles

Transduction of Tolerogenic Dendritic Cells by Foamy Virus and Lentivirus Vectors.

RuiJun Su, MD, Ph.D., Neil C. Josephson, MD

Blood (2004) 104 (11): 1356.

https://doi.org/10.1182/blood.V104.11.1356.1356

Published: 2004

Journal Articles

Gene transfer into murine hematopoietic stem cells with helper-free foamy virus vectors

George Vassilopoulos, Grant Trobridge, Neil C. Josephson, David W. Russell

Blood (2001) 98 (3): 604–609.

https://doi.org/10.1182/blood.V98.3.604

Published: 2001

NARROW

BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression

Results of Genetic Analysis of 11,341 Participants Enrolled in the My Life, Our Future (MLOF) Hemophilia Genotyping Initiative

Real-World Clinical and Economic Burden of Hematopoietic Cell Transplantation Among a Large US Commercially Insured Populationhematopoietic CELL Transplantation Among a Large US Commercially Insured Population

Toxicity Burden of Bleomycin Treatment in Hodgkin Lymphoma: A Systematic Literature Review

Novel Approach to and Results of Genetic Analysis of 3000 Hemophilia Patients Enrolled in the MyLifeOurFuture Initiative

Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A

Phosphorylation Of The Cytoplasmic Tyrosine Residues Of c-Mpl Is Not Required To Preserve Intact Receptor Dependent Hematopoietic Stem Cell Survival Signaling

Safety and prolonged activity of recombinant factor VIII Fc fusion protein in hemophilia A patients

Recombinant factor IX-Fc fusion protein (rFIXFc) demonstrates safety and prolonged activity in a phase 1/2a study in hemophilia B patients

Determination of Anti-Factor VIIII Antibody Titers, Isotypes and Epitopes by SPR Analysis of Human Plasma

Factor VIII Mutation and Desmopressin-Responsiveness in 63 Patients with Mild Hemophilia A

Survey of Current Prophylaxis Practices and Bleeding Characteristics of Children with Severe Hemophilia A In U.S. Hemophilia Treatment Centers.

Incomplete restoration of Mpl expression in the mpl −/− mouse produces partial correction of the stem cell–repopulating defect and paradoxical thrombocytosis

Induction of Tolerance to FVIII in Hemophilic Mice by Delivery of Apoptotic Syngeneic Fibroblasts Expressing a FVIII Transgene.

Reduction of Anti-FVIII Inhibitor Titers in Hemophilic Mice Infused with Syngeneic Apoptotic Cells Expressing a Human FVIII Transgene.

Transduction of Primate Cells with Feline Foamy Virus Envelope Pseudotyped Prototype Foamy Virus Vectors.

Transduction of Tolerogenic Dendritic Cells by Foamy Virus and Lentivirus Vectors.

Gene transfer into murine hematopoietic stem cells with helper-free foamy virus vectors

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American Society of Hematology

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American Society of Hematology

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