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Journal Articles

Lentiglobin Gene Therapy for Patients with Transfusion-Dependent β-Thalassemia (TDT): Results from the Phase 3 Northstar-2 and Northstar-3 Studies

Franco Locatelli, Mark C. Walters, MD, Janet L. Kwiatkowski, MD, John Porter, MA MDFRCP FRCPath, Martin G. Sauer, MD, Isabelle Thuret, MD, Suradej Hongeng, MD, Andreas E. Kulozik, MD PhD, Ashutosh Lal, MD, Adrian J. Thrasher, MD PhD FMedSci, Evangelia Yannaki, MD, Heidi Elliot, Ge Tao, PhD, Mohammed Asmal, MD PhD, Alexis A. Thompson, MD MPH

Blood (2018) 132 (Supplement 1): 1025.

https://doi.org/10.1182/blood-2018-99-112667

Published: 2018

Journal Articles

Results from the Hgb-207 (Northstar-2) Trial: A Phase 3 Study to Evaluate Safety and Efficacy of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia (TDT) in Patients with Non-β 0 /β 0 Genotypes

Mark C. Walters, MD, Suradej Hongeng, MD, Janet L. Kwiatkowski, MD, Franco Locatelli, MD PhD, John B. Porter, BA MB BChir MRCP(Path) FRCP(Path) MD, Martin G. Sauer, Adrian J Thrasher, MD PhD FMedSci, Isabelle Thuret, MD, Evangelia Yannaki, MD PhD, Heidi Elliot, Marisa Gayron, Mohammed Asmal, MD PhD, Alexis A Thompson, MD MPH

Blood (2017) 130 (Supplement 1): 526.

https://doi.org/10.1182/blood.V130.Suppl_1.526.526

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

Diverse and Distinct Human Hematopoietic Differentiation Programs Generate By Bone Marrow and G-CSF Mobilized Hematopoietic Stem and Progenitors Cells

Blood (2017) 130 (Supplement 1): 4618.

https://doi.org/10.1182/blood.V130.Suppl_1.4618.4618

Published: 2017

Journal Articles

Automated Manufacture of CAR19 Donor Lymphocytes Incorporated into an Escalated Dose DLI Protocol: Preliminary Results from the CARD Study

Blood (2017) 130 (Supplement 1): 1993.

https://doi.org/10.1182/blood.V130.Suppl_1.1993.1993

Published: 2017

Journal Articles

a Diversity of Human Hematopoietic Differentiation Programs Identified through In Vivo Tracking of Hematopoiesis in Wiskott-Aldrich Syndrome Patients

Emmanuelle Six, PhD, Laure Caccavelli, Arnaud Lecoules, Christopher L Nobles, PhD, France Male, Cécile Roudaut, MSc, Clemence Plantier, Alessandra Magnani, MD PhD, Elisa Magrin, PhD, Nicolas Cagnard, Anne Galy, PharmD, PhD, Adrian J Thrasher, MD PhD, Salima hacein-Bey Abina, Agathe Guilloux, Isabelle Andre-Schmutz, Charles C. Berry, frederic D. Bushman, Marina Cavazzana, MD PhD

Blood (2016) 128 (22): 3871.

https://doi.org/10.1182/blood.V128.22.3871.3871

Published: 2016

Journal Articles

Preclinical Development of Gene Therapy for X-Linked Severe Combined Immunodeficiency (SCID-X1)

Valentina Poletti, PhD, Sabine Charrier, PhD, Samia Martin, Bernard Gjata, Alban Vignaud, PhD, Fang Zhang, PhD, Karen Buckland, PhD, Michael Rothe, PhD, Axel Schambach, MD PhD, Sung-Yun Pai, MD, David A. Williams, MD, H. Bobby Gaspar, MD PhD, Adrian J Thrasher, MD PhD, Fulvio Mavilio, PhD

Blood (2016) 128 (22): 4705.

https://doi.org/10.1182/blood.V128.22.4705.4705

Published: 2016

Journal Articles

New Molecular Surrogate Assay for Genotoxicity Assessment of Gene Therapy Vectors (SAGA)

Adrian Schwarzer, MD PhD, Steven R Talbot, PhD, Oliver Dittrich-Breiholz, PhD, Adrian J Thrasher, MD PhD, Bobby Gaspar, MD, Ute Modlich, Christopher Baum, MD, Axel Schambach, MD PhD, Michael Rothe, PhD

Blood (2016) 128 (22): 4710.

https://doi.org/10.1182/blood.V128.22.4710.4710

Published: 2016

Journal Articles

First Clinical Application of Talen Engineered Universal CAR19 T Cells in B-ALL

Waseem Qasim, MBBS PhD, Persis Jal Amrolia, Sujith Samarasinghe, MD PhD, Sara Ghorashian, MD PhD FRCPath, Hong Zhan, PhD, Sian Stafford, PhD, Katie Butler, PhD, Gul Ahsan, Kimberly Gilmour, Stuart Adams, PhD, Danielle Pinner, Robert Chiesa, Steve Chatters, PhD, Sue Swift, PhD, Nicholas Goulden, MD PhD, Karl Peggs, MBBChir, MRCP, FRCPath, Adrian J Thrasher, MD PhD, Paul Veys, Martin Pule, PhD

Blood (2015) 126 (23): 2046.

https://doi.org/10.1182/blood.V126.23.2046.2046

Published: 2015

Journal Articles

Correction of Hemoglobin Levels in a Heterozygous Humanized Mouse Model of Thalassemia after Fetal Gene Therapy

Panicos Shangaris, MDMSc, Stavros P Loukogeorgakis, MDBSc PhDMRCS(Eng.), Sindhu Subramaniam, PhD, Mike Blundell, PhD, Nahla Bakhamis, MSc, Shanrun Liu, MD PhD, Simon Eaton, PhD, Durrgah Ramachandra, MSc, Panayiotis Maghsoudlou, BSc, Luca Urbani, PhD, Simon Waddington, MSc, PhD, Joy Archer, VMD PhDFRCPath DECVCP MRCVS, Mike Antoniou, PhD, Adrian J Thrasher, MD PhD, Thomas Ryan, PhD, Paolo De Coppi, MDPhD, Anna David, MDPhDMRCOG

Blood (2014) 124 (21): 3495.

https://doi.org/10.1182/blood.V124.21.3495.3495

Published: 2014

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

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

CD26 Inhibition Can Aid the Homing of Cytokine Activated Mobilized Peripheral Blood (MPB )CD34 + Cells to the Bone Marrow (BM) but a Ligand Dependent Attachment Defect Prevents Their Long Term Retention and Subsequent Engraftment

Konstantina Kallinikou, Fernando Anjos-Afonso, Michael P Blundell, Stuart J Ings, Deepika Kassen, PhD, Adrian J Thrasher, MD, PhD, David C Linch, MD FMedSci, Dominique Bonnet, PhD, Kwee Yong, PhD

Blood (2011) 118 (21): 141.

https://doi.org/10.1182/blood.V118.21.141.141

Published: 2011

Journal Articles

The Clonal Inventory of Gene Corrected Hematopoiesis in Three Successful Clinical Gene Therapy Trials

Kerstin Schwarzwaelder, PhD, Manfred Schmidt, PhD, Annette Deichmann, Steven J. Howe, PhD, Marion G. Ott, MD, Stefan Stein, PhD, Ulrich Siler, MD, Salima Hacein-Bey-Abina, PhD, Hanno Glimm, MD, Dieter Hoelzer, MD, Marina Cavazzana-Calvo, MD, PhD, Adrian J. Thrasher, MD, PhD, Reinhard Seger, MD, Manuel Grez, PhD, Christof von Kalle, MD

Blood (2007) 110 (11): 3733.

https://doi.org/10.1182/blood.V110.11.3733.3733

Published: 2007

Journal Articles

Non-Random Integration and Clone Selection by Lentiviral SIN-LTR Vectors.

Cynthia C. Bartholomae, Annette Deichmann, Manfred Schmidt, PhD, Rafael J. Yanez-Munoz, PhD, Steven J. Howe, PhD, Sonja Schmidt, Claudia Prinz, Adrian J. Thrasher, MD, PhD, Christof von Kalle, MD

Blood (2006) 108 (11): 3257.

https://doi.org/10.1182/blood.V108.11.3257.3257

Published: 2006

NARROW

Lentiglobin Gene Therapy for Patients with Transfusion-Dependent β-Thalassemia (TDT): Results from the Phase 3 Northstar-2 and Northstar-3 Studies

Results from the Hgb-207 (Northstar-2) Trial: A Phase 3 Study to Evaluate Safety and Efficacy of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia (TDT) in Patients with Non-β 0 /β 0 Genotypes

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

Diverse and Distinct Human Hematopoietic Differentiation Programs Generate By Bone Marrow and G-CSF Mobilized Hematopoietic Stem and Progenitors Cells

Automated Manufacture of CAR19 Donor Lymphocytes Incorporated into an Escalated Dose DLI Protocol: Preliminary Results from the CARD Study

a Diversity of Human Hematopoietic Differentiation Programs Identified through In Vivo Tracking of Hematopoiesis in Wiskott-Aldrich Syndrome Patients

Preclinical Development of Gene Therapy for X-Linked Severe Combined Immunodeficiency (SCID-X1)

New Molecular Surrogate Assay for Genotoxicity Assessment of Gene Therapy Vectors (SAGA)

First Clinical Application of Talen Engineered Universal CAR19 T Cells in B-ALL

Correction of Hemoglobin Levels in a Heterozygous Humanized Mouse Model of Thalassemia after Fetal Gene Therapy

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

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

CD26 Inhibition Can Aid the Homing of Cytokine Activated Mobilized Peripheral Blood (MPB )CD34 + Cells to the Bone Marrow (BM) but a Ligand Dependent Attachment Defect Prevents Their Long Term Retention and Subsequent Engraftment

The Clonal Inventory of Gene Corrected Hematopoiesis in Three Successful Clinical Gene Therapy Trials

Non-Random Integration and Clone Selection by Lentiviral SIN-LTR Vectors.

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American Society of Hematology

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ASH Publications

American Society of Hematology

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