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Journal Articles

Gene Therapy for Fanconi Anemia [Group A]: Interim Results of RP-L102 Clinical Trials

Blood (2021) 138 (Supplement 1): 3968.

https://doi.org/10.1182/blood-2021-147071

Published: 2021

Journal Articles

Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: Interim Results of a Global Phase 1 Study for Adult and Pediatric Patients

Blood (2021) 138 (Supplement 1): 563.

https://doi.org/10.1182/blood-2021-148161

Published: 2021

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): Interim Results

Donald B. Kohn, Claire Booth, Julián Sevilla, Gayatri R Rao, Elena Almarza, Dayna Terrazas, Eileen Nicoletti, Augustine Fernandes, Caroline Kuo, Satiro de Oliveira, Theodore B. Moore, Kenneth M Law, Brian C Beard, Grace Choi, Miriam Zeini, Cristina Mesa-Núñez, Adrian J. Thrasher, Juan A Bueren, Jonathan D Schwartz

Blood (2021) 138 (Supplement 1): 2932.

https://doi.org/10.1182/blood-2021-151898

Published: 2021

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

Intravenous injection of a foamy virus vector to correct canine SCID-X1

Christopher R. Burtner, Brian C. Beard, Douglas R. Kennedy, Martin E. Wohlfahrt, Jennifer E. Adair, Grant D. Trobridge, Andrew M. Scharenberg, Troy R. Torgerson, David J. Rawlings, Peter J. Felsburg, Hans-Peter Kiem

Blood (2014) 123 (23): 3578–3584.

https://doi.org/10.1182/blood-2013-11-538926

Published: 2014

Includes: Supplemental data

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

Positive selection of mC46-expressing CD4 + T cells and maintenance of virus specific immunity in a primate AIDS model

Patrick M. Younan, Patricia Polacino, John P. Kowalski, Christopher W. Peterson, Nicholas J. Maurice, Nathaniel P. Williams, On Ho, Grant D. Trobridge, Dorothee Von Laer, Martin Prlic, Brian C. Beard, Stephen DeRosa, Shiu-Lok Hu, Hans-Peter Kiem

Blood (2013) 122 (2): 179–187.

https://doi.org/10.1182/blood-2013-01-482224

Published: 2013

Includes: Supplemental data

Journal Articles

Efficient generation, purification, and expansion of CD34 + hematopoietic progenitor cells from nonhuman primate–induced pluripotent stem cells

Jennifer L. Gori, Devikha Chandrasekaran, John P. Kowalski, Jennifer E. Adair, Brian C. Beard, Sunita L. D'Souza, Hans-Peter Kiem

Blood (2012) 120 (13): e35–e44.

https://doi.org/10.1182/blood-2012-05-433797

Published: 2012

Includes: Supplemental data

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

Long-term polyclonal and multilineage engraftment of methylguanine methyltransferase P140K gene-modified dog hematopoietic cells in primary and secondary recipients

Brian C. Beard, Reeteka Sud, Kirsten A. Keyser, Christina Ironside, Tobias Neff, Sabine Gerull, Grant D. Trobridge, Hans-Peter Kiem

Blood (2009) 113 (21): 5094–5103.

https://doi.org/10.1182/blood-2008-09-176412

Published: 2009

Includes: Supplemental data

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

NARROW

Gene Therapy for Fanconi Anemia [Group A]: Interim Results of RP-L102 Clinical Trials

Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: Interim Results of a Global Phase 1 Study for Adult and Pediatric Patients

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

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

Intravenous injection of a foamy virus vector to correct canine SCID-X1

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

Positive selection of mC46-expressing CD4 + T cells and maintenance of virus specific immunity in a primate AIDS model

Efficient generation, purification, and expansion of CD34 + hematopoietic progenitor cells from 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.

Long-term polyclonal and multilineage engraftment of methylguanine methyltransferase P140K gene-modified dog hematopoietic cells in primary and secondary recipients

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

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American Society of Hematology

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American Society of Hematology

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