Hubert B. Gaspar

UCL Great Ormond Street Institute Of Child Health
London, ENG WC1N1EH, United Kingdom

Last Updated: 09/26/2026

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Found 97 publications

Anti-CD45 PBD-based antibody-drug conjugates are effective targeted conditioning agents for gene therapy and stem cell transplant.

Anti-CD45 PBD-based antibody-drug conjugates are effective targeted conditioning agents for gene therapy and stem cell transplant.

Journal: Molecular therapy : the journal of the American Society of Gene Therapy

Published: April 28, 2026

GAA engineered to improve cellular uptake enhances correction in a preclinical hematopoietic stem cell gene therapy model of Pompe disease.

GAA engineered to improve cellular uptake enhances correction in a preclinical hematopoietic stem cell gene therapy model of Pompe disease.

Journal: Molecular therapy. Advances

Published: February 06, 2026

Advances in Gene Therapy for Inherited Haemoglobinopathies.

Advances in Gene Therapy for Inherited Haemoglobinopathies.

Journal: Hematology reports

Published: November 24, 2025

Long-Term Safety and Efficacy of Gene Therapy for Adenosine Deaminase Deficiency.

Long-Term Safety and Efficacy of Gene Therapy for Adenosine Deaminase Deficiency.

Journal: The New England journal of medicine

Published: October 15, 2025

Anti-CD45 PBD-based antibody-drug conjugates are effective targeted conditioning agents for gene therapy and stem cell transplant.

Anti-CD45 PBD-based antibody-drug conjugates are effective targeted conditioning agents for gene therapy and stem cell transplant.

Journal: Molecular therapy : the journal of the American Society of Gene Therapy

Published: July 27, 2023

Lentiviral Gene Transfer Corrects Immune Abnormalities in XIAP Deficiency.

Lentiviral Gene Transfer Corrects Immune Abnormalities in XIAP Deficiency.

Journal: Journal of clinical immunology

Published: August 22, 2022

Author Correction: Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott-Aldrich syndrome.

Author Correction: Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott-Aldrich syndrome.

Journal: Nature medicine

Published: August 09, 2022

Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier.

Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier.

Journal: Molecular therapy : the journal of the American Society of Gene Therapy

Published: June 22, 2022

Retrospective, Landmark Analysis of Long-term Adult Morbidity Following Allogeneic HSCT for Inborn Errors of Immunity in Infancy and Childhood.

Retrospective, Landmark Analysis of Long-term Adult Morbidity Following Allogeneic HSCT for Inborn Errors of Immunity in Infancy and Childhood.

Journal: Journal of clinical immunology

Published: December 21, 2021

Author Correction: Metabolite and thymocyte development defects in ADA-SCID mice receiving enzyme replacement therapy.

Author Correction: Metabolite and thymocyte development defects in ADA-SCID mice receiving enzyme replacement therapy.

Journal: Scientific reports

Published: December 16, 2021

Metabolite and thymocyte development defects in ADA-SCID mice receiving enzyme replacement therapy.

Metabolite and thymocyte development defects in ADA-SCID mice receiving enzyme replacement therapy.

Journal: Scientific reports

Published: September 27, 2021

Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency.

Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency.

Journal: The New England journal of medicine

Published: May 11, 2021

Showing 1-12 of 97

Last Updated: 09/26/2026