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Journal Articles

Development of Anti-CD45 Antibody Drug Conjugates As Targeted Conditioning Agents for Transplantation/Gene Therapy with Potent Anti-Leukemic Properties

Jenny Yeung, Aiyin Liao, Matthew Shaw, Soraia Caetano-Silva, Winston Vetharoy, Ian Kirby, Francesca Zammarchi, Karin Havenith, Patrick H van Berkel, Claire Booth, H Bobby Gaspar, Adrian J. Thrasher, Kerry Chester, Persis J Amrolia

Blood (2022) 140 (Supplement 1): 10211–10212.

https://doi.org/10.1182/blood-2022-162390

Published: 2022

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

Clinical and immunological features in a cohort of patients with partial DiGeorge syndrome followed at a single center

Giuliana Giardino, Nesrine Radwan, Patra Koletsi, Deborah M. Morrogh, Stuart Adams, Winnie Ip, Austen Worth, Alison Jones, Imke Meyer-Parsonson, H. Bobby Gaspar, Kimberly Gilmour, E. Graham Davies, Fani Ladomenou

Blood (2019) 133 (24): 2586–2596.

https://doi.org/10.1182/blood.2018885244

Published: 2019

Journal Articles

G-CSF Vs Haematopoietic Stem Cell Transplantation in Severe Congenital Neutropenia with ELANE Mutation: Role of G-CSF Dose. a Retrospective Controlled Study on Behalf of Saawp (Severe Aplastic Anemia Working Party) of the EBMT, of the Stem Cell Transplant for Immunodeficiency Group in Europe (SCETIDE), of the Severe Chronic Neutropenia French Registry (SCNFR) and Italian Neutropenia Registry (INR)

Blood (2017) 130 (Supplement 1): 2267.

https://doi.org/10.1182/blood.V130.Suppl_1.2267.2267

Published: 2017

Journal Articles

Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult

Blood (2017) 130 (11): 1327–1335.

https://doi.org/10.1182/blood-2017-04-777136

Published: 2017

Includes: Supplemental data

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

Stem cell transplantation in severe congenital neutropenia: an analysis from the European Society for Blood and Marrow Transplantation

Blood (2015) 126 (16): 1885–1892.

https://doi.org/10.1182/blood-2015-02-628859

Published: 2015

Journal Articles

Tearing RAGs apart

H. Bobby Gaspar

Blood (2014) 123 (2): 156–157.

https://doi.org/10.1182/blood-2013-11-535633

Published: 2014

Journal Articles

How I treat severe combined immunodeficiency

H. Bobby Gaspar, Waseem Qasim, E. Graham Davies, Kanchan Rao, Persis J. Amrolia, Paul Veys

Blood (2013) 122 (23): 3749–3758.

https://doi.org/10.1182/blood-2013-02-380105

Published: 2013

Journal Articles

Outcomes Of Haematopoietic Stem Cell Transplantation (HSCT) for Severe Congenital Neutropenia (SCN): Preliminary Results

Blood (2013) 122 (21): 3355.

https://doi.org/10.1182/blood.V122.21.3355.3355

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

SAP gene transfer restores cellular and humoral immune function in a murine model of X-linked lymphoproliferative disease

Christine Rivat, Claire Booth, Maria Alonso-Ferrero, Michael Blundell, Neil J. Sebire, Adrian J. Thrasher, H. Bobby Gaspar

Blood (2013) 121 (7): 1073–1076.

https://doi.org/10.1182/blood-2012-07-445858

Published: 2013

Includes: Supplemental data

Journal Articles

Gene Therapy for Hemophagocytic Lymphohistiocytosis (HLH): Fixing a Criticial ‘Circuit Breaker’ in the Immune System.

Michael Jordan, MD, Marlene Carmo, PhD, Swati Tiwari, Paritha Arumugam, PhD, Kimberly Risma, MD PhD, Bobby Gaspar, MD, Punam Malik, MD

Blood (2012) 120 (21): 3158.

https://doi.org/10.1182/blood.V120.21.3158.3158

Published: 2012

Journal Articles

Gene therapy for ADA-SCID: defining the factors for successful outcome

H. Bobby Gaspar

Blood (2012) 120 (18): 3628–3629.

https://doi.org/10.1182/blood-2012-08-446559

Published: 2012

Journal Articles

Outcome of hematopoietic stem cell transplantation for adenosine deaminase–deficient severe combined immunodeficiency

Blood (2012) 120 (17): 3615–3624.

https://doi.org/10.1182/blood-2011-12-396879

Published: 2012

Includes: Supplemental data

Journal Articles

Transplantation in patients with SCID: mismatched related stem cells or unrelated cord blood?

Blood (2012) 119 (12): 2949–2955.

https://doi.org/10.1182/blood-2011-06-363572

Published: 2012

Includes: Supplemental data

Journal Articles

Dyskeratosis Congenita: Evaluation of Immune Status and Hematopoietic Stem Cell Transplantation. A Literature and EBMT Data Base Survey of 75 Patients,

Elisabeth T Korthof, MD, Judith Van der Zalm, med. student, Martine F Raphael, MD, Dorine Bresters, MD, PhD, Jakub Tolar, MD, PhD, Mahmoud Aljurf, MD, Paul Veys, MD MRCP FRCPath, Rosi Oneto, Andrea Bacigalupo, MD, Christina Peters, MD, Bobby Gaspar, MD, Carlo Dufour, MD, Judith C. W. Marsh, Prof MD PhD

Blood (2011) 118 (21): 4144.

https://doi.org/10.1182/blood.V118.21.4144.4144

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

Neonatal diagnosis of severe combined immunodeficiency leads to significantly improved survival outcome: the case for newborn screening

Lucinda Brown, Jinhua Xu-Bayford, Zoe Allwood, Mary Slatter, Andrew Cant, E. Graham Davies, Paul Veys, Andrew R. Gennery, H. Bobby Gaspar

Blood (2011) 117 (11): 3243–3246.

https://doi.org/10.1182/blood-2010-08-300384

Published: 2011

NARROW

Development of Anti-CD45 Antibody Drug Conjugates As Targeted Conditioning Agents for Transplantation/Gene Therapy with Potent Anti-Leukemic Properties

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

Clinical and immunological features in a cohort of patients with partial DiGeorge syndrome followed at a single center

Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult

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)

Stem cell transplantation in severe congenital neutropenia: an analysis from the European Society for Blood and Marrow Transplantation

Tearing RAGs apart

How I treat severe combined immunodeficiency

Outcomes Of Haematopoietic Stem Cell Transplantation (HSCT) for Severe Congenital Neutropenia (SCN): Preliminary Results

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

SAP gene transfer restores cellular and humoral immune function in a murine model of X-linked lymphoproliferative disease

Gene Therapy for Hemophagocytic Lymphohistiocytosis (HLH): Fixing a Criticial ‘Circuit Breaker’ in the Immune System.

Gene therapy for ADA-SCID: defining the factors for successful outcome

Outcome of hematopoietic stem cell transplantation for adenosine deaminase–deficient severe combined immunodeficiency

Transplantation in patients with SCID: mismatched related stem cells or unrelated cord blood?

Dyskeratosis Congenita: Evaluation of Immune Status and Hematopoietic Stem Cell Transplantation. A Literature and EBMT Data Base Survey of 75 Patients,

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

Neonatal diagnosis of severe combined immunodeficiency leads to significantly improved survival outcome: the case for newborn screening

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American Society of Hematology

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American Society of Hematology

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