Publications by authors named "Bobby Gaspar"

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Long-Term Safety and Efficacy of Gene Therapy for Adenosine Deaminase Deficiency.
Claire Booth, Katelyn Masiuk, Konstantinos Vazouras, Augustine Fernandes, Jinhua Xu-Bayford, H Bobby Gaspar

N Engl J Med· October 2025

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Anti-CD45 PBD-based antibody-drug conjugates are effective targeted conditioning agents for gene therapy and stem cell transplant.
Jenny Yeung, Aiyin Liao, Matthew Shaw, Soraia Silva, Winston Vetharoy, H Bobby Gaspar

Mol Ther· June 2024

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The transformative potential of HSC gene therapy as a genetic medicine.
Pervinder Sagoo, H Bobby Gaspar

Gene Ther· April 2023

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Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier.
Bethan J Critchley, H Bobby Gaspar, Sara Benedetti

Mol Ther· March 2023

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Lentiviral Gene Transfer Corrects Immune Abnormalities in XIAP Deficiency.
Joseph Topal, Neelam Panchal, Amairelys Barroeta, Anna Roppelt, Annelotte Mudde, H Bobby Gaspar

J Clin Immunol· February 2023

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Retrospective, Landmark Analysis of Long-term Adult Morbidity Following Allogeneic HSCT for Inborn Errors of Immunity in Infancy and Childhood.
James W Day, Reem Elfeky, Bethany Nicholson, Rupert Goodman, Rachel Pearce, H Bobby Gaspar

J Clin Immunol· August 2022

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Long-Term Immune Recovery After Hematopoietic Stem Cell Transplantation for ADA Deficiency: a Single-Center Experience.
Alexandra Y Kreins, Helena F Velasco, Kai-Ning Cheong, Kanchan Rao, Paul Veys, H Bobby Gaspar

J Clin Immunol· January 2022

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Metabolite and thymocyte development defects in ADA-SCID mice receiving enzyme replacement therapy.
Federico A Moretti, Giuliana Giardino, Teresa C H Attenborough, Athina Soragia Gkazi, Ben K Margetts, H Bobby Gaspar

Sci Rep· December 2021

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Author Correction: Metabolite and thymocyte development defects in ADA-SCID mice receiving enzyme replacement therapy.
Federico A Moretti, Giuliana Giardino, Teresa C H Attenborough, Athina Soragia Gkazi, Ben K Margetts, H Bobby Gaspar

Sci Rep· December 2021

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Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency.
Donald B Kohn, Claire Booth, Kit L Shaw, Jinhua Xu-Bayford, Elizabeth Garabedian, H Bobby Gaspar

N Engl J Med· May 2021

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Long-term lymphoid progenitors independently sustain naïve T and NK cell production in humans.
Natalia Izotova, Christine Rivat, Cristina Baricordi, Elena Blanco, Danilo Pellin, H Bobby Gaspar

Nat Commun· March 2021

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Busulfan Pharmacokinetics in Adenosine Deaminase-Deficient Severe Combined Immunodeficiency Gene Therapy.
Kathryn L Bradford, Siyu Liu, Maja Krajinovic, Marc Ansari, Elizabeth Garabedian, H Bobby Gaspar

Biol Blood Marrow Transplant· October 2020

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Lentiviral Hematopoietic Stem Cell Gene Therapy Rescues Clinical Phenotypes in a Murine Model of Pompe Disease.
Giuseppa Piras, Claudia Montiel-Equihua, Yee-Ka Agnes Chan, Slawomir Wantuch, Daniel Stuckey, H Bobby Gaspar

Mol Ther Methods Clin Dev· September 2020

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Successful Preclinical Development of Gene Therapy for Recombinase-Activating Gene-1-Deficient SCID.
Laura Garcia-Perez, Marja van Eggermond, Lieke van Roon, Sandra A Vloemans, Martijn Cordes, H Bobby Gaspar

Mol Ther Methods Clin Dev· June 2020

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Lentiviral gene therapy for X-linked chronic granulomatous disease.
Donald B Kohn, Claire Booth, Elizabeth M Kang, Sung-Yun Pai, Kit L Shaw, H Bobby Gaspar

Nat Med· February 2020

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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, H Bobby Gaspar

Blood· June 2019

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Pre-clinical Safety and Efficacy of Lentiviral Vector-Mediated Stem Cell Gene Therapy for the Treatment of Mucopolysaccharidosis IIIA.
Stuart M Ellison, Aiyin Liao, Shaun Wood, Jessica Taylor, Amir Saam Youshani, H Bobby Gaspar

Mol Ther Methods Clin Dev· June 2019

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Consensus approach for the management of severe combined immune deficiency caused by adenosine deaminase deficiency.
Donald B Kohn, Michael S Hershfield, Jennifer M Puck, Alessandro Aiuti, Annaliesse Blincoe, H Bobby Gaspar

J Allergy Clin Immunol· March 2019

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T-cell gene therapy for perforin deficiency corrects cytotoxicity defects and prevents hemophagocytic lymphohistiocytosis manifestations.
Sujal Ghosh, Marlene Carmo, Miguel Calero-Garcia, Ida Ricciardelli, Juan Carlos Bustamante Ogando, H Bobby Gaspar

J Allergy Clin Immunol· September 2018

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Transfer of gene-corrected T cells corrects humoral and cytotoxic defects in patients with X-linked lymphoproliferative disease.
Neelam Panchal, Ben Houghton, Begona Diez, Sujal Ghosh, Ida Ricciardelli, H Bobby Gaspar

J Allergy Clin Immunol· July 2018

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Umbilical cord blood transplantation without in vivo T-cell depletion for children with MHC class II deficiency.
Reem Elfeky, Juliana M Furtado-Silva, Robert Chiesa, Kanchan Rao, Giovanna Lucchini, Bobby Gaspar

J Allergy Clin Immunol· June 2018

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Non-Clinical Efficacy and Safety Studies on G1XCGD, a Lentiviral Vector for Ex Vivo Gene Therapy of X-Linked Chronic Granulomatous Disease.
Christian Brendel, Michael Rothe, Giorgia Santilli, Sabine Charrier, Stefan Stein, Bobby Gaspar

Hum Gene Ther Clin Dev· June 2018

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Preclinical Development of a Lentiviral Vector for Gene Therapy of X-Linked Severe Combined Immunodeficiency.
Valentina Poletti, Sabine Charrier, Guillaume Corre, Bernard Gjata, Alban Vignaud, H Bobby Gaspar

Mol Ther Methods Clin Dev· June 2018

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The 2017 IUIS Phenotypic Classification for Primary Immunodeficiencies.
Aziz Bousfiha, Leïla Jeddane, Capucine Picard, Fatima Ailal, H Bobby Gaspar

J Clin Immunol· January 2018

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International Union of Immunological Societies: 2017 Primary Immunodeficiency Diseases Committee Report on Inborn Errors of Immunity.
Capucine Picard, H Bobby Gaspar, Waleed Al-Herz, Aziz Bousfiha, Jean-Laurent Casanova

J Clin Immunol· January 2018

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