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Journal Articles

Adeno-Associated Mediated Gene Transfer for Hemophilia B:8 Year Follow up and Impact of Removing "Empty Viral Particles" on Safety and Efficacy of Gene Transfer

Amit C Nathwani, MD PhD, Ulrike Reiss, MD, Edward Tuddenham, MD, Pratima Chowdary, MD, Jenny McIntosh, PhD, Anne Riddell, Jun Pie, Johnny N Mahlangu, MD, Michael Recht, MD PhD, Yu-Min Shen, MD, Kathleen G. Halka, MD, Michael M Meagher, PhD, Arthur W. Nienhuis, MD, Andrew M. Davidoff, MD, Sarah Mangles, Chris L Morton, Zhou Junfang, Vlad Calin Radulescu, MD

Blood (2018) 132 (Supplement 1): 491.

https://doi.org/10.1182/blood-2018-99-118334

Published: 2018

Journal Articles

Blood ’s 70th anniversary: 1988-1992

Arthur W. Nienhuis

Blood (2016) 127 (9): 1075–1076.

https://doi.org/10.1182/blood-2015-10-638668

Published: 2016

Journal Articles

Cellular Immune Responses To Vector In a Gene Therapy Trial For Hemophilia B Using An AAV8 Self-Complementary Factor IX Vector

Etiena Basner-Tschakarjan, MD PhD, Federico Mingozzi, PhD, Yifeng Chen, MD, Amit Nathwani, MD, PhD, Edward Tuddenham, MD, Anne Riddell, BSc, Catherine Ng, MS, Pradip Rustagi, MD, Bertil Glader, MD, PhD, Mark Kay, MD, PhD, Ulrike M. Reiss, MD, Arthur W. Nienhuis, MD, Andrew Davidoff, MD, Katherine A. High, MD

Blood (2013) 122 (21): 717.

https://doi.org/10.1182/blood.V122.21.717.717

Published: 2013

Journal Articles

Development of gene therapy for blood disorders: an update

Arthur W. Nienhuis

Blood (2013) 122 (9): 1556–1564.

https://doi.org/10.1182/blood-2013-04-453209

Published: 2013

Journal Articles

Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant

Blood (2013) 121 (17): 3335–3344.

https://doi.org/10.1182/blood-2012-10-462200

Published: 2013

Includes: Supplemental data

Journal Articles

Stable Factor IX Activity Following AAV-Mediated Gene Transfer in Patients with Severe Hemophilia B

Blood (2012) 120 (21): 752.

https://doi.org/10.1182/blood.V120.21.752.752

Published: 2012

Journal Articles

Adeno-Associated Viral Vector Mediated Gene Transfer for Hemophilia B

Blood (2011) 118 (21): 5.

https://doi.org/10.1182/blood.V118.21.5.5

Published: 2011

Journal Articles

Somatic Gene Therapy for X-Linked Severe Combined Immunodeficiency Using a Self-Inactivating Modified Gammaretroviral Vector Results in An Improved Preclinical Safety Profile and Early Clinical Efficacy in a Human Patient

Blood (2011) 118 (21): 164.

https://doi.org/10.1182/blood.V118.21.164.164

Published: 2011

Journal Articles

Transcriptional regulation of fetal to adult hemoglobin switching: new therapeutic opportunities

Andrew Wilber, Arthur W. Nienhuis, Derek A. Persons

Blood (2011) 117 (15): 3945–3953.

https://doi.org/10.1182/blood-2010-11-316893

Published: 2011

Journal Articles

Therapeutic levels of fetal hemoglobin in erythroid progeny of β-thalassemic CD34 + cells after lentiviral vector-mediated gene transfer

Andrew Wilber, Phillip W. Hargrove, Yoon-Sang Kim, Janice M. Riberdy, Vijay G. Sankaran, Eleni Papanikolaou, Maria Georgomanoli, Nicholas P. Anagnou, Stuart H. Orkin, Arthur W. Nienhuis, Derek A. Persons

Blood (2011) 117 (10): 2817–2826.

https://doi.org/10.1182/blood-2010-08-300723

Published: 2011

Includes: Supplemental data

Journal Articles

Early Clinical Trial Results Following Administration of a Low Dose of a Novel Self Complementary Adeno-Associated Viral Vector Encoding Human Factor IX In Two Subjects with Severe Hemophilia B

Amit Nathwani, MD, PhD, Edward Tuddenham, MD, Cecilia Rosales, Jenny McIntosh, Anne Riddell, Pradip Rustagi, Bertil Glader, Mark Kay, MD, PhD, James Allay, PhD, John Coleman, Susan Sleep, Katherine A. High, MD, Federico Mingozzi, PhD., John T. Gray, PhD, Ulrike M. Reiss, MD, Arthur W. Nienhuis, MD, Andrew Davidoff

Blood (2010) 116 (21): 248.

https://doi.org/10.1182/blood.V116.21.248.248

Published: 2010

Journal Articles

A zinc-finger transcriptional activator designed to interact with the γ-globin gene promoters enhances fetal hemoglobin production in primary human adult erythroblasts

Andrew Wilber, Ulrich Tschulena, Phillip W. Hargrove, Yoon-Sang Kim, Derek A. Persons, Carlos F. Barbas, III, Arthur W. Nienhuis

Blood (2010) 115 (15): 3033–3041.

https://doi.org/10.1182/blood-2009-08-240556

Published: 2010

Includes: Supplemental data

Journal Articles

A Zinc-Finger Transcriptional Activator Designed to Interact with the Gamma-Globin Gene Promoters Enhances Fetal Hemoglobin Production in Erythroid Cells Derived From Normal and Beta-Thalassemic CD34 + Cells.

Andrew Wilber, PhD, Uli Tschulena, PhD, Phillip W. Hargrove, MS, Yoon-Sang Kim, MD, PhD, Carlos F. Barbas, III, PhD, Derek A. Persons, MD, PhD, Arthur W. Nienhuis, MD

Blood (2009) 114 (22): 3567.

https://doi.org/10.1182/blood.V114.22.3567.3567

Published: 2009

Journal Articles

Lentiviral Vector-Mediated Transfer of the Gamma-Globin Gene Into Normal and Beta-Thalassemic Human CD34 + Cells Results in Potentially Therapeutic Levels of Fetal Hemoglobin in Erythroid Progeny.

Andrew Wilber, Ph.D., Phillip W. Hargrove, M.S., Yoon-Sang Kim, MD, PhD, Arthur W. Nienhuis, M.D., Derek A. Persons, MD, PhD

Blood (2009) 114 (22): 3579.

https://doi.org/10.1182/blood.V114.22.3579.3579

Published: 2009

Journal Articles

Sustained high-level polyclonal hematopoietic marking and transgene expression 4 years after autologous transplantation of rhesus macaques with SIV lentiviral vector–transduced CD34 + cells

Yoo-Jin Kim, Yoon-Sang Kim, Andre Larochelle, Gabriel Renaud, Tyra G. Wolfsberg, Rima Adler, Robert E. Donahue, Peiman Hematti, Bum-Kee Hong, Jean Roayaei, Keiko Akagi, Janice M. Riberdy, Arthur W. Nienhuis, Cynthia E. Dunbar, Derek A. Persons

Blood (2009) 113 (22): 5434–5443.

https://doi.org/10.1182/blood-2008-10-185199

Published: 2009

Includes: Supplemental data

Journal Articles

Development of gene therapy for blood disorders

Arthur W. Nienhuis

Blood (2008) 111 (9): 4431–4444.

https://doi.org/10.1182/blood-2007-11-078121

Published: 2008

Journal Articles

An experimental system for the evaluation of retroviral vector design to diminish the risk for proto-oncogene activation

Byoung Y. Ryu, Marguerite V. Evans-Galea, John T. Gray, David M. Bodine, Derek A. Persons, Arthur W. Nienhuis

Blood (2008) 111 (4): 1866–1875.

https://doi.org/10.1182/blood-2007-04-085506

Published: 2008

Includes: Supplemental data

Journal Articles

The Effect of a Chromatin Insulator (5′cHS4) on Enhanced Expression of the LMO2 Proto-Oncogene by an Oncoretroviral Long Terminal Repeat.

Byoung Y. Ryu, PhD, John T. Gray, PhD, David M. Bodine, PhD, Arthur W. Nienhuis, MD

Blood (2006) 108 (11): 3253.

https://doi.org/10.1182/blood.V108.11.3253.3253

Published: 2006

Journal Articles

Long-Term Polyclonal and Stable Gene Transfer into Rhesus Repopulating Hematopoietic Stem Cells Using a Simian Immunodeficiency Virus-Based Lentiviral Vector System.

Yoo-Jin Kim, MD, Nadia L. Hussein, Peiman Hematti, MD, Bum-Kee Hong, MD, Boris Calmels, PhD, Robert E. Donahue, DVM, Hideki Hanawa, MD, Arthur W. Nienhuis, MD, Cynthia E. Dunbar, MD

Blood (2006) 108 (11): 3259.

https://doi.org/10.1182/blood.V108.11.3259.3259

Published: 2006

Journal Articles

Acute myeloid leukemia is associated with retroviral gene transfer to hematopoietic progenitor cells in a rhesus macaque

Ruth Seggewiss, Stefania Pittaluga, Rima L. Adler, F. Javier Guenaga, Cole Ferguson, Ingo H. Pilz, Byoung Ryu, Brian P. Sorrentino, W. Scott Young, III, Robert E. Donahue, Christof von Kalle, Arthur W. Nienhuis, Cynthia E. Dunbar

Blood (2006) 107 (10): 3865–3867.

https://doi.org/10.1182/blood-2005-10-4108

Published: 2006

Includes: Supplemental data

NARROW

Adeno-Associated Mediated Gene Transfer for Hemophilia B:8 Year Follow up and Impact of Removing "Empty Viral Particles" on Safety and Efficacy of Gene Transfer

Blood ’s 70th anniversary: 1988-1992

Cellular Immune Responses To Vector In a Gene Therapy Trial For Hemophilia B Using An AAV8 Self-Complementary Factor IX Vector

Development of gene therapy for blood disorders: an update

Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant

Stable Factor IX Activity Following AAV-Mediated Gene Transfer in Patients with Severe Hemophilia B

Adeno-Associated Viral Vector Mediated Gene Transfer for Hemophilia B

Somatic Gene Therapy for X-Linked Severe Combined Immunodeficiency Using a Self-Inactivating Modified Gammaretroviral Vector Results in An Improved Preclinical Safety Profile and Early Clinical Efficacy in a Human Patient

Transcriptional regulation of fetal to adult hemoglobin switching: new therapeutic opportunities

Therapeutic levels of fetal hemoglobin in erythroid progeny of β-thalassemic CD34 + cells after lentiviral vector-mediated gene transfer

Early Clinical Trial Results Following Administration of a Low Dose of a Novel Self Complementary Adeno-Associated Viral Vector Encoding Human Factor IX In Two Subjects with Severe Hemophilia B

A zinc-finger transcriptional activator designed to interact with the γ-globin gene promoters enhances fetal hemoglobin production in primary human adult erythroblasts

A Zinc-Finger Transcriptional Activator Designed to Interact with the Gamma-Globin Gene Promoters Enhances Fetal Hemoglobin Production in Erythroid Cells Derived From Normal and Beta-Thalassemic CD34 + Cells.

Lentiviral Vector-Mediated Transfer of the Gamma-Globin Gene Into Normal and Beta-Thalassemic Human CD34 + Cells Results in Potentially Therapeutic Levels of Fetal Hemoglobin in Erythroid Progeny.

Sustained high-level polyclonal hematopoietic marking and transgene expression 4 years after autologous transplantation of rhesus macaques with SIV lentiviral vector–transduced CD34 + cells

Development of gene therapy for blood disorders

An experimental system for the evaluation of retroviral vector design to diminish the risk for proto-oncogene activation

The Effect of a Chromatin Insulator (5′cHS4) on Enhanced Expression of the LMO2 Proto-Oncogene by an Oncoretroviral Long Terminal Repeat.

Long-Term Polyclonal and Stable Gene Transfer into Rhesus Repopulating Hematopoietic Stem Cells Using a Simian Immunodeficiency Virus-Based Lentiviral Vector System.

Acute myeloid leukemia is associated with retroviral gene transfer to hematopoietic progenitor cells in a rhesus macaque

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American Society of Hematology

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American Society of Hematology

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