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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
Journal: Blood
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

Donald B. Kohn, MD, Kit L. Shaw, PhD, Elizabeth Garabedian, RN, MS, Denise Ann Carbonaro-Sarracino, PhD, Theodore B. Moore, MD, Satiro N. De Oliveira, MD, Gay M. Crooks, MD, John Tse, Sally Shupien, Dayna Terrazas, Alejandra Davila, Amalia Icreverzi, PhD, Allen Yu, Krista M. Chun, Christian E. Casas, Provaboti Barman, PhD, Maritess Coronel, Beatriz Campo Fernandez, PhD, Ruixue Zhang, Roger P. Hollis, PhD, Chilenwa Uzowuru, Hilory Ricketts, Jinhua Xu Bayford, Valentina Trevisan, Serena Arduini, PhD, Frances Lynn, Mahesh Kudari, MBBS, MA, Andrea Spezzi, MD MBBS, Lilith Reeves, MS, MT(ASCP), Kenneth Cornetta, Robert A. Sokolic, MD, Roberta Parrott, BS, Rebecca Buckley, MD, Claire Booth, Fabio Candotti, MD, Harry L. Malech, MD, Adrian J. Thrasher, MBBS, PhD FMedSci, H. Bobby Gaspar, MBBS, PhD
Journal: Blood
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
Journal: Blood
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)

Francesca Fioredda, MD, Filomena Pierri, Sabrina Zanardi, Bobby Gaspar, Phil Ancliff, Marc Bierings, MD PhD, Gulyuz Ozturk, Owen Smith, MD, Paul Veys, MD PhD, Per Ljungman, Regis Peffault De Latour, MD PhD, Reuven Or, MD, Robert Wynn, MD, Krzysztof Kalwak, MD, Giovanna Russo, MD, Fabio Tucci, MD, Christine M. Knoll, MD, Baldo Martire, Claudio Favre, MD, Christine bellanne Chantelot, MDPhD, Blandine Beaupain, MsC, Jean Donadieu, MD, Carlo Dufour, MD
Journal: Blood
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

Clinical Trials & Observations
Emma C. Morris, Thomas Fox, Ronjon Chakraverty, Rita Tendeiro, Katie Snell, Christine Rivat, Sarah Grace, Kimberly Gilmour, Sarita Workman, Karen Buckland, Katie Butler, Ronnie Chee, Alan D. Salama, Hazem Ibrahim, Havinder Hara, Cecile Duret, Fulvio Mavilio, Frances Male, Frederick D. Bushman, Anne Galy, Siobhan O. Burns, H. Bobby Gaspar, Adrian J. Thrasher
Journal: Blood
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
Journal: Blood
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
Journal: Blood
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

CME Clinical Trials & Observations
Francesca Fioredda, Simona Iacobelli, Anja van Biezen, Bobby Gaspar, Phil Ancliff, Jean Donadieu, Mahmoud Aljurf, Christina Peters, Michaela Calvillo, Susanne Matthes-Martin, Giuseppe Morreale, Nelleke van ’t Veer-Tazelaar, Liesbeth de Wreede, Amal Al Seraihy, Akif Yesilipek, Alain Fischer, Marc Bierings, Gulyuz Ozturk, Owen Smith, Paul Veys, Per Ljungman, Régis Peffault de Latour, José Sánchez de Toledo Codina, Reuven Or, Arnold Ganser, Boris Afanasyev, Robert Wynn, Krzysztof Kalwak, Judith Marsh, Carlo Dufour
Journal: Blood
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
Journal: Blood
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
Journal: Blood
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

Francesca Fioredda, Liesbeth C de Wreede, Michaela Calvillo, Bobby Gaspar, Mahmoud Aljurf, Anja van Biezen, Giuseppe Morreale, Christina Peters, Erica M Knoedler, Amal Al-Seraihy, M. Akif Yesilipek, Alain Fisher, Marc Bierings, Jean Donadieu, Gulyuz Ozturk, Francoise Bourge, Victoria Bordon, Owen Smith, Andrea Bacigalupo, Jakob Passweg, Gerard Socie, Hubert Shretzenmeier, Judith Marsh, Carlo Dufour, MD
Journal: Blood
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

Salima Hacein-Bey-Abina, Pharm.D., Ph.D., Sung-Yun Pai, MD, Frederic Bushman, PhD, Myriam Armant, PhD, Stephane Blanche, MD, Johanna Blondeau, Laure Caccavelli, Heather Daley, BS, Colleen Dansereau, RN, BSN, CPN, CPHON, Satiro deOliveira, MD, Dongjing Guo, MPH, Gregory Hopkins, BS, Annick Lim, M.Sci, Wendy B. London, PHD, Nirav Malani, Frances Male, BA, Punam Malik, MD, Maria Angelica Marinovic, MD, Sean McDonough, MS, Matias Oleastro, MD, Capucine Picard, MD PhD, Jerome Ritz, MD, Axel Schambach, MD PhD, Leslie E Silberstein, MD, Alla Tsytsykova, PhD, Johannes C.M. Van der Loo, PhD, Christopher Baum, MD, H. Bobby Gaspar, MD PhD, Donald B. Kohn, MD, Alexandra H. Filipovich, MD, Luigi Daniele Notarangelo, MD, Alain Fischer, MD, Marina Cavazzana-Calvo, MD, PhD, David A. Williams, MD, Adrian J Thrasher, MD, PhD
Journal: Blood
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

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

CME Clinical Trials & Observations
Amel Hassan, Claire Booth, Alex Brightwell, Zoe Allwood, Paul Veys, Kanchan Rao, Manfred Hönig, Wilhelm Friedrich, Andrew Gennery, Mary Slatter, Robbert Bredius, Andrea Finocchi, Caterina Cancrini, Alessandro Aiuti, Fulvio Porta, Arnalda Lanfranchi, Michela Ridella, Colin Steward, Alexandra Filipovich, Rebecca Marsh, Victoria Bordon, Saleh Al-Muhsen, Hamoud Al-Mousa, Zobaida Alsum, Hasan Al-Dhekri, Abdulaziz Al Ghonaium, Carsten Speckmann, Alain Fischer, Nizar Mahlaoui, Kim E. Nichols, Eyal Grunebaum, Daifulah Al Zahrani, Chaim M. Roifman, Jaap Boelens, E. Graham Davies, Marina Cavazzana-Calvo, Luigi Notarangelo, H. Bobby Gaspar
Journal: Blood
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?

Clinical Trials & Observations
Juliana F. Fernandes, Vanderson Rocha, Myriam Labopin, Benedicte Neven, Despina Moshous, Andrew R. Gennery, Wilhelm Friedrich, Fulvio Porta, Cristina Diaz de Heredia, Donna Wall, Yves Bertrand, Paul Veys, Mary Slatter, Ansgar Schulz, Ka Wah Chan, Michael Grimley, Mouhab Ayas, Tayfun Gungor, Wolfram Ebell, Carmem Bonfim, Krzysztof Kalwak, Pierre Taupin, Stéphane Blanche, H. Bobby Gaspar, Paul Landais, Alain Fischer, Eliane Gluckman, Marina Cavazzana-Calvo
Journal: 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
Journal: Blood
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

Sung-Yun Pai, MD, Luigi Daniele Notarangelo, MD, Chad Harris, Federica Cattaneo, MD, Matthew Wladkowski, Myriam Armant, PhD, Grace S. Kao, MD, Matthew Wielgosz, Arthur W. Nienhuis, MD, Ute Modlich, DVM, PhD, Martijn Brugman, PhD, Axel Schambach, MD PhD, Michael Rothe, Susannah I Thornhill, PhD, H. Bobby Gaspar, Kathryn Parsley, PhD, Samantha Cooray, PhD, Elke Grassman, Ph.D., Frederic Bushman, PhD, Sally Shupien, Matias Oleastro, MD, Salima Hacein-Bey-Abina, Alain Fischer, MD, Marina Cavazzana-Calvo, MD, PhD, Punam Malik, MD, Alexandra Filipovich, MD, Donald B. Kohn, MD, Christopher Baum, MD, David A. Williams, MD, Adrian J Thrasher, MD, PhD
Journal: Blood
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

Brief Report
Lucinda Brown, Jinhua Xu-Bayford, Zoe Allwood, Mary Slatter, Andrew Cant, E. Graham Davies, Paul Veys, Andrew R. Gennery, H. Bobby Gaspar
Journal: Blood
Blood (2011) 117 (11): 3243–3246.
https://doi.org/10.1182/blood-2010-08-300384
Published: 2011