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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

Investigating Zeta Globin Gene Expression to Develop a Potential Therapy for Alpha Thalassemia Major

Georgia L. Gregory, BS, Beeke Wienert, PhD, Marisa Schwab, MD, Billie Rachael Lianoglou, MS, Roger P. Hollis, PhD, Donald B. Kohn, MD, Bruce R. Conklin, MD, Tippi C. MacKenzie, MD

Blood (2020) 136 (Supplement 1): 3–4.

https://doi.org/10.1182/blood-2020-142922

Published: 2020

Journal Articles

First Report of Non-Genotoxic Conditioning with JSP191 (anti-CD117) and Hematopoietic Stem Cell Transplantation in a Newly Diagnosed Patient with Severe Combined Immune Deficiency

Blood (2020) 136 (Supplement 1): 10.

https://doi.org/10.1182/blood-2020-137762

Published: 2020

Journal Articles

Lentiviral Gene Therapy with Autologous Hematopoietic Stem and Progenitor Cells (HSPCs) for the Treatment of Severe Combined Immune Deficiency Due to Adenosine Deaminase Deficiency (ADA-SCID): Results in an Expanded Cohort

Blood (2019) 134 (Supplement_1): 3345.

https://doi.org/10.1182/blood-2019-123432

Published: 2019

Journal Articles

Notch Signaling Promotes the Differentiation of Human CLEC9A+ Dendritic Cells from Hematopoietic Stem and Progenitor Cells

Christopher S. Seet, Suwen Li, Brent Chick, David Casero, Eric H. Gschweng, Yuhua Zhu, Runfeng Miao, Amelie Montel-Hagen, PhD, Donald B Kohn, MD, Mary Sehl, Gay M. Crooks, MD

Blood (2017) 130 (Supplement 1): 624.

https://doi.org/10.1182/blood.V130.Suppl_1.624.624

Published: 2017

Journal Articles

Leukocyte Adhesion Deficiency-I (LAD-I): A Comprehensive Review of Published Cases

Sanchali Kasbekar, PharmD, Juan Bueren, PhD, Adrian J Thrasher, MD PhD FMedSci, Donald B Kohn, MD, Julian Sevilla, MD PhD, Jonathan D Schwartz, MD, Elena Almarza, PhD

Blood (2017) 130 (Supplement 1): 4821.

https://doi.org/10.1182/blood.V130.Suppl_1.4821.4821

Published: 2017

Journal Articles

Development of a Treg Lineage Specific Lentiviral Vector for the Treatment of IPEX Syndrome

Katelyn E Masiuk, BS, Jennifer Laborada, BS, Roger P Hollis, PhD, Donald B Kohn, MD

Blood (2017) 130 (Supplement 1): 528.

https://doi.org/10.1182/blood.V130.Suppl_1.528.528

Published: 2017

Journal Articles

A Pre-Clinical Model Of Hematopoietic Stem Cell Based Immunotherapy For Cancer Utilizing The NY-ESO-1 T-Cell Receptor and sr39TK PET Reporter / Suicide Gene

Eric H. Gschweng, BS, Melissa N. McCracken, BS, Thinle Chodon, MD, PhD, Richard C Koya, MD, PhD, Michael L Kaufman, MS, Michelle Ho, BS, Navdeep Saini, Roger P. Hollis, PhD, Antoni Ribas, MD, PhD, Satiro N. De Oliveira, MD, Owen N. Witte, MD, Donald B. Kohn, MD

Blood (2013) 122 (21): 2020.

https://doi.org/10.1182/blood.V122.21.2020.2020

Published: 2013

Journal Articles

Zinc Finger Nucleases Targeting The β-Globin Locus Drive Efficient Correction Of The Sickle Mutation In CD34 + Cells

Megan D Hoban, BS, Alok V Joglekar, PhD, David Gray, BS, Michael L Kaufman, MS, Fabrizia Urbinati, PhD, Shantha Senadheera, MS, Gregory Cost, PhD, Andreas Reik, PhD, Michael C. Holmes, PhD, Philip D Gregory, PhD, Roger P. Hollis, PhD, Donald B. Kohn, MD

Blood (2013) 122 (21): 2904.

https://doi.org/10.1182/blood.V122.21.2904.2904

Published: 2013

Journal Articles

Anti-CD19 Chimeric Antigen Receptor Controlled By The Suicide Gene HSVsr39TK In Hematopoietic Stem Cells For Immunotherapy Of B-Lineage Malignancies

Sarah M. Larson, MD, Andy Tu, Shanta Senadheera, Michelle Ho, BS, Donald B. Kohn, MD, Satiro deOliveira, MD

Blood (2013) 122 (21): 1659.

https://doi.org/10.1182/blood.V122.21.1659.1659

Published: 2013

Journal Articles

Immune Reconstitution and Preliminary Safety Analysis Of 9 Patients Treated With Somatic Gene Therapy For X-Linked Severe Combined Immunodeficiency (SCID-X1) With a Self-Inactivating Gammaretroviral Vector

Blood (2013) 122 (21): 715.

https://doi.org/10.1182/blood.V122.21.715.715

Published: 2013

Journal Articles

Inducing Definitive Erythropoiesis From Human Embryonic Stem Cells Through a Novel Intracellular MPL Dimerization Strategy

William Sang Kim, B.S., Yuhua Zhu, B.S., Qiming Deng, B.S., Amanda J. Grieco, B.S., Gautam G. Dravid, Ph.D, Chintan Parekh, MBBS, Roger P. Hollis, PhD, Timothy Lane, Ph.D, Eric E. Bouhassira, PhD, Donald B. Kohn, MD, Gay M. Crooks, MBBS

Blood (2013) 122 (21): 1172.

https://doi.org/10.1182/blood.V122.21.1172.1172

Published: 2013

Journal Articles

Efficient Erythropoiesis From Human Embryonic Stem Cells Through Dimerization of Intracellular MPL.

William Sang Kim, B.S., Gautam G. Dravid, Ph.D, Yuhua Zhu, B.S., Chintan Parekh, MBBS, Qiming Deng, B.S., Roger P. Hollis, PhD, Timothy Lane, Ph.D, Donald B. Kohn, MD, Gay M. Crooks, MBBS

Blood (2012) 120 (21): 2291.

https://doi.org/10.1182/blood.V120.21.2291.2291

Published: 2012

Journal Articles

Increasing Transduction Efficiency in Human Hematopoietic Stem Cells for Gene Therapy

Kismet M Baldwin, MD, Fabrizia Urbinati, Ph.D, Zulema Romero-Garcia, Ph D, Donald B. Kohn, MD

Blood (2012) 120 (21): 2052.

https://doi.org/10.1182/blood.V120.21.2052.2052

Published: 2012

Journal Articles

Anti-CD117 (c-Kit) Monoclonal Antibodies Deplete Human Hematopoietic Stem Cells and Facilitate Their Replacement in Humanized NOD/SCID/IL2Rγ −/− Mice: A Non-Toxic Conditioning Regimen for Allotransplantation

Aaron C. Logan, MD, PhD, Agnieszka Czechowicz, MD, PhD, Benjamin V. Kelley, BS, Theingi M. Thway, PhD, Ivan Magana, Mark R. Krampf, MS, Jessica Poyser, Roger P. Hollis, PhD, Donald B. Kohn, MD, Irving L. Weissman, MD, Judith A. Shizuru, MD, PhD

Blood (2012) 120 (21): 4099.

https://doi.org/10.1182/blood.V120.21.4099.4099

Published: 2012

Journal Articles

Gene Transfer to Hematopoietic Stem/Progenitor Cells As a Novel Approach for Immunotherapy Against B-Lineage Malignancies: In Vivo Xenograft Model,

Satiro N. De Oliveira, MD, Francesca Giannoni, Ph.D., Cinnamon Hardee, MS, Arineh Sahaghian, Laurence J N Cooper, MD, PhD, Donald B. Kohn, MD

Blood (2011) 118 (21): 4168.

https://doi.org/10.1182/blood.V118.21.4168.4168

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

Laser Tweezers Raman Spectroscopy As a Novel Red Blood Cell Functional Assay for Sickle Cell Disease

Rui Liu, Lena Zheng, Denis M. Dwyre, MD, Fabrizia Urbinati, Ph.D, Donald B. Kohn, MD, Dennis Matthews, James Chan, PhD, Noriko Satake, MD

Blood (2011) 118 (21): 4847.

https://doi.org/10.1182/blood.V118.21.4847.4847

Published: 2011

Journal Articles

Preclinical Studies for Sickle Cell Disease Gene Therapy Using Bone Marrow CD34+ Cells Modified with a βAS3-Globin Lentiviral Vector

Fabrizia Urbinati, Ph.D, Zulema Romero Garcia, Ph.D, Sabine Geiger, Rafael Ruiz de Assin, Ph.D, Gabriela Kuftinec, Roger P. Hollis, PhD, Satiro N. De Oliveira, MD, Jennifer Wherley, Aaron Cooper, Tim M. Townes, PhD, Gay M. Crooks, MBBS, Elizabeth J Read, MD, Mark C. Walters, MD, Herbert J. Meiselman, ScD, Thomas D. Coates, MD, Victor J. Marder, M.D., Donald B. Kohn, MD

Blood (2011) 118 (21): 3119.

https://doi.org/10.1182/blood.V118.21.3119.3119

Published: 2011

Journal Articles

CD19 Fc-Fusion Protein for Detection of Cells Expressing Anti-CD19 Chimeric Antigen Receptors.

Satiro N. De Oliveira, MD, Jiexin Wang, MD, Roger P. Hollis, PhD, Donald B. Kohn, MD

Blood (2010) 116 (21): 3756.

https://doi.org/10.1182/blood.V116.21.3756.3756

Published: 2010

NARROW

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

Investigating Zeta Globin Gene Expression to Develop a Potential Therapy for Alpha Thalassemia Major

First Report of Non-Genotoxic Conditioning with JSP191 (anti-CD117) and Hematopoietic Stem Cell Transplantation in a Newly Diagnosed Patient with Severe Combined Immune Deficiency

Lentiviral Gene Therapy with Autologous Hematopoietic Stem and Progenitor Cells (HSPCs) for the Treatment of Severe Combined Immune Deficiency Due to Adenosine Deaminase Deficiency (ADA-SCID): Results in an Expanded Cohort

Notch Signaling Promotes the Differentiation of Human CLEC9A+ Dendritic Cells from Hematopoietic Stem and Progenitor Cells

Leukocyte Adhesion Deficiency-I (LAD-I): A Comprehensive Review of Published Cases

Development of a Treg Lineage Specific Lentiviral Vector for the Treatment of IPEX Syndrome

A Pre-Clinical Model Of Hematopoietic Stem Cell Based Immunotherapy For Cancer Utilizing The NY-ESO-1 T-Cell Receptor and sr39TK PET Reporter / Suicide Gene

Zinc Finger Nucleases Targeting The β-Globin Locus Drive Efficient Correction Of The Sickle Mutation In CD34 + Cells

Anti-CD19 Chimeric Antigen Receptor Controlled By The Suicide Gene HSVsr39TK In Hematopoietic Stem Cells For Immunotherapy Of B-Lineage Malignancies

Immune Reconstitution and Preliminary Safety Analysis Of 9 Patients Treated With Somatic Gene Therapy For X-Linked Severe Combined Immunodeficiency (SCID-X1) With a Self-Inactivating Gammaretroviral Vector

Inducing Definitive Erythropoiesis From Human Embryonic Stem Cells Through a Novel Intracellular MPL Dimerization Strategy

Efficient Erythropoiesis From Human Embryonic Stem Cells Through Dimerization of Intracellular MPL.

Increasing Transduction Efficiency in Human Hematopoietic Stem Cells for Gene Therapy

Anti-CD117 (c-Kit) Monoclonal Antibodies Deplete Human Hematopoietic Stem Cells and Facilitate Their Replacement in Humanized NOD/SCID/IL2Rγ −/− Mice: A Non-Toxic Conditioning Regimen for Allotransplantation

Gene Transfer to Hematopoietic Stem/Progenitor Cells As a Novel Approach for Immunotherapy Against B-Lineage Malignancies: In Vivo Xenograft Model,

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

Laser Tweezers Raman Spectroscopy As a Novel Red Blood Cell Functional Assay for Sickle Cell Disease

Preclinical Studies for Sickle Cell Disease Gene Therapy Using Bone Marrow CD34+ Cells Modified with a βAS3-Globin Lentiviral Vector

CD19 Fc-Fusion Protein for Detection of Cells Expressing Anti-CD19 Chimeric Antigen Receptors.

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American Society of Hematology

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ASH Publications

American Society of Hematology

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