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Journal Articles

Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: A Global Phase 1 Study for Adult and Pediatric Patients

Blood (2020) 136 (Supplement 1): 47.

https://doi.org/10.1182/blood-2020-137246

Published: 2020

Journal Articles

Gene Therapy for Fanconi Anemia, Complementation Group a: Updated Results from Ongoing Global Clinical Studies of RP-L102

Agnieszka Czechowicz, MDPhD, Rajni Agarwal, MD, Julián Sevilla, MD PhD, Paula Río, PhD, Susana Navarro, PhD, Brian C Beard, PhD, Kenneth M Law, PhD, Grace Choi, BS, Miriam Zeini, PhD, Eileen Nicoletti, MD, John E Wagner, MD, Claire Booth, MBBS, PhD MSc, Jonathan D Schwartz, MD, Juan A Bueren, PhD, Maria Grazia Roncarolo, MD

Blood (2020) 136 (Supplement 1): 14.

https://doi.org/10.1182/blood-2020-137106

Published: 2020

Journal Articles

A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Results from Phase 1

Donald B. Kohn, MD, Gayatri R Rao, MD JD, Elena Almarza, PhD, Dayna Terrazas, RN, Eileen Nicoletti, MD, Augustine Fernandes, PhD, Caroline Kuo, MD, Satiro N. De Oliveira, MD, Theodore B. Moore, MD, Kenneth M Law, PhD, Brian C Beard, PhD, Julian Sevilla, MD PhD, Cristina Mesa-Núñez, PhD, Juan A Bueren, PhD, Jonathan D Schwartz, MD

Blood (2020) 136 (Supplement 1): 15.

https://doi.org/10.1182/blood-2020-142484

Published: 2020

Journal Articles

Gene Therapy for Lad-I Immunodeficiency: Preclinical Evaluation of HSC Transduction Under Optimized GMP-Conditions

Elena Almarza, PhD, Cristina Mesa-Núñez, PhD, Carlos Damián, María Fernández-García, Begoña Díez-Cabezas, PhD, M Luz Lozano, Paula Rio, PhD, Rosa M Yañez, PhD, Ken Law, PhD, Brian C Beard, PhD, Jonathan D Schwartz, MD, Juan A. Bueren, PhD

Blood (2019) 134 (Supplement_1): 5751.

https://doi.org/10.1182/blood-2019-122878

Published: 2019

Journal Articles

Changing the Natural History of Fanconi Anemia Complementation Group-A with Gene Therapy: Early Results of U.S. Phase I Study of Lentiviral-Mediated Ex-Vivo FANCA Gene Insertion in Human Stem and Progenitor Cells

Agnieszka Czechowicz, MD PhD, Maria Grazia Roncarolo, MD, Brian C Beard, PhD, Ken Law, PhD, Eileen Nicoletti, MD, Paula Río, PhD, Juan A. Bueren, PhD, Jonathan D Schwartz, MD, Sandeep Soni, MBBS

Blood (2019) 134 (Supplement_1): 3350.

https://doi.org/10.1182/blood-2019-127352

Published: 2019

Journal Articles

Safety Of a Gamma Globin Expressing Lentivirus Vector In a Non-Human Primate Model For Gene Therapy Of Sickle Cell Disease

Hans-Peter Kiem, MD, Paritha Arumugam, PhD, Burtner Christopher, PhD, Jennifer E Adair, PhD, Brian C Beard, PhD, Catherine Fox, MS, Punam Malik, MD

Blood (2013) 122 (21): 2896.

https://doi.org/10.1182/blood.V122.21.2896.2896

Published: 2013

Journal Articles

In Vivo Selection and Long-Term Engraftment Of Hematopoietic Stem Cells Generated Via Vascular Niche Induction Of Nonhuman Primate Induced Pluripotent Stem Cells

Jennifer L Gori, PhD, Jason M Butler, PhD, Devikha Chandrasekaran, MS, Brian C Beard, PhD, Daniel J Nolan, PhD, Michael Ginsberg, PhD, Jennifer E Adair, PhD, Shahin Rafii, MD, Hans-Peter Kiem, MD

Blood (2013) 122 (21): 466.

https://doi.org/10.1182/blood.V122.21.466.466

Published: 2013

Journal Articles

Drug Resistance Gene Therapy to Augment Allogeneic Transplantation for the Treatment of Leukemia Following Relapse

Melanie Wurm, DVM, PhD, Nathaniel Williams, BS, Christina Ironside, BS, Brian C Beard, PhD, Hans-Peter Kiem, MD

Blood (2011) 118 (21): 2050.

https://doi.org/10.1182/blood.V118.21.2050.2050

Published: 2011

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

Towards Defining An Optimal Conditioning Regimen for Stem Cell Gene Therapy In Fanconi Anemia.

Pamela S. Becker, MD, PhD, Jennifer Adair, PhD, Xin Zhao, MD, Sylvia Chien, Martin E Wohlfahrt, PhD, Grant Trobridge, PhD, Brian C Beard, PhD, Hans-Peter Kiem

Blood (2010) 116 (21): 1478.

https://doi.org/10.1182/blood.V116.21.1478.1478

Published: 2010

Journal Articles

Efficient MGMTP140K-Mediated In Vivo Selection and Chemoprotection of Long-Term Repopulating Cells in Nonhuman Primates Following Reduced Intensity Conditioning.

Brian C Beard, PhD, Grant D Trobridge, PhD, Jeannine S McCune, Hans-Peter Kiem, MD

Blood (2009) 114 (22): 3572.

https://doi.org/10.1182/blood.V114.22.3572.3572

Published: 2009

Journal Articles

Correction of Fanconi Anemia Group A in Primary Human and Murine Hematopoietic Progenitors with a Clinical Lentiviral Vector

Jason Taylor, MD, PhD, Grant D Trobridge, PhD, Martin Wohlfahrt, Sylvia Chien, Xin Zhao, MD, Brian C Beard, PhD, Hans-Peter Kiem, MD, Pamela S. Becker, MD, PhD

Blood (2008) 112 (11): 2357.

https://doi.org/10.1182/blood.V112.11.2357.2357

Published: 2008

Journal Articles

Efficient Gene Marking with a Clinical Gammaretrovirus Vector Expressing MGMTP140K in Baboons and Macaques

Brian C Beard, PhD, Megan L Welsh, MS, Grant D Trobridge, PhD, Hans-Peter Kiem, MD

Blood (2008) 112 (11): 3535.

https://doi.org/10.1182/blood.V112.11.3535.3535

Published: 2008

Journal Articles

Sustained Stable Engraftment of MGMT Gene-Modified Long-Term Repopulating Cells in Primary and Secondary Canine Recipients.

Brian C. Beard, PhD, Reeteka Sud, PhD, Sabine Gerull, MD, Laura J. Peterson, BS, Tobias Neff, MD, Hans-Peter Kiem, MD

Blood (2006) 108 (11): 202.

https://doi.org/10.1182/blood.V108.11.202.202

Published: 2006

Journal Articles

Development of Leukemia after HOXB4 Gene Transfer in the Canine Model.

Xiao-Bing Zhang, PhD, Brian C. Beard, PhD, Grant Trobridge, PhD, Robert Hackman, Eileen Bryant, R. Keith Humphries, MD, PhD, Hans-Peter Kiem, MD

Blood (2006) 108 (11): 204.

https://doi.org/10.1182/blood.V108.11.204.204

Published: 2006

Journal Articles

Unique Integration Profiles of Gammaretrovirus, Lentivirus, and Foamy Virus Transduced Dog Long-Term Repopulating Cells.

Kirsten A. Keyser, Brian C. Beard, PhD, Grant Trobridge, PhD, Laura J. Peterson, Dan Miller, PhD, Mike Jacobs, PhD, Rajinder Kaul, PhD, Hans-Peter Kiem, MD

Blood (2006) 108 (11): 3252.

https://doi.org/10.1182/blood.V108.11.3252.3252

Published: 2006

Journal Articles

MGMT (P140K) Allows for Efficient and Sustained In Vivo Selection in Non-Human Primates.

Brian C. Beard, PhD, Kate Beebe, BS, Julia Piasecki, BS, Christina Gooch, BS, David Dickerson, BS, Hans-Peter Kiem, MD

Blood (2006) 108 (11): 3270.

https://doi.org/10.1182/blood.V108.11.3270.3270

Published: 2006

Journal Articles

Differential Effects of HOXB4 Overexpression on Short and Long-Term Repopulating Cells in Nonhuman Primates.

Xiao-Bing Zhang, PhD, Brian C. Beard, PhD, Katherine Beebe, R. Keith Humphries, PhD, MD, Hans-Peter Kiem, MD

Blood (2005) 106 (11): 33.

https://doi.org/10.1182/blood.V106.11.33.33

Published: 2005

Journal Articles

Genomic Mapping of Retrovirus Integration Profiles after In Vivo Selection of Chemo-Protected Stem Cells in the Clinically Relevant Canine Model.

Brian C. Beard, PhD, Pau Mezquita, MD, PhD, Grant Trobridge, PhD, Laura J. Peterson, MS, Tobias Neff, MD, Alixandra A. Knapp, BA, Hans-Peter Kiem, MD

Blood (2005) 106 (11): 1293.

https://doi.org/10.1182/blood.V106.11.1293.1293

Published: 2005

Journal Articles

Molecular Characterization of Stem Cell Populations and Specific Clone Tracking of Retrovirally-Marked Chemoprotected Stem Cells before and after Dose-Escalating Chemotherapy.

Brian C. Beard, PhD, Pau Mezquita, MD, PhD, Laura J. Peterson, BS, Tobias Neff, MD, Ponni Anandakumar, MS, Alixandra A. Knapp, BA, Hans-Peter Kiem, MD

Blood (2004) 104 (11): 291.

https://doi.org/10.1182/blood.V104.11.291.291

Published: 2004

NARROW

Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: A Global Phase 1 Study for Adult and Pediatric Patients

Gene Therapy for Fanconi Anemia, Complementation Group a: Updated Results from Ongoing Global Clinical Studies of RP-L102

A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Results from Phase 1

Gene Therapy for Lad-I Immunodeficiency: Preclinical Evaluation of HSC Transduction Under Optimized GMP-Conditions

Changing the Natural History of Fanconi Anemia Complementation Group-A with Gene Therapy: Early Results of U.S. Phase I Study of Lentiviral-Mediated Ex-Vivo FANCA Gene Insertion in Human Stem and Progenitor Cells

Safety Of a Gamma Globin Expressing Lentivirus Vector In a Non-Human Primate Model For Gene Therapy Of Sickle Cell Disease

In Vivo Selection and Long-Term Engraftment Of Hematopoietic Stem Cells Generated Via Vascular Niche Induction Of Nonhuman Primate Induced Pluripotent Stem Cells

Drug Resistance Gene Therapy to Augment Allogeneic Transplantation for the Treatment of Leukemia Following Relapse

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

Towards Defining An Optimal Conditioning Regimen for Stem Cell Gene Therapy In Fanconi Anemia.

Efficient MGMTP140K-Mediated In Vivo Selection and Chemoprotection of Long-Term Repopulating Cells in Nonhuman Primates Following Reduced Intensity Conditioning.

Correction of Fanconi Anemia Group A in Primary Human and Murine Hematopoietic Progenitors with a Clinical Lentiviral Vector

Efficient Gene Marking with a Clinical Gammaretrovirus Vector Expressing MGMTP140K in Baboons and Macaques

Sustained Stable Engraftment of MGMT Gene-Modified Long-Term Repopulating Cells in Primary and Secondary Canine Recipients.

Development of Leukemia after HOXB4 Gene Transfer in the Canine Model.

Unique Integration Profiles of Gammaretrovirus, Lentivirus, and Foamy Virus Transduced Dog Long-Term Repopulating Cells.

MGMT (P140K) Allows for Efficient and Sustained In Vivo Selection in Non-Human Primates.

Differential Effects of HOXB4 Overexpression on Short and Long-Term Repopulating Cells in Nonhuman Primates.

Genomic Mapping of Retrovirus Integration Profiles after In Vivo Selection of Chemo-Protected Stem Cells in the Clinically Relevant Canine Model.

Molecular Characterization of Stem Cell Populations and Specific Clone Tracking of Retrovirally-Marked Chemoprotected Stem Cells before and after Dose-Escalating Chemotherapy.

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American Society of Hematology

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ASH Publications

American Society of Hematology

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