Adrian J. Thrasher

Great Ormond Street
London, ENG, United Kingdom

Last Updated: 09/26/2026

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Found 244 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

A modified cyclosporine enhances lentivector transduction ex vivo and in vivo by degrading IFITM3.

A modified cyclosporine enhances lentivector transduction ex vivo and in vivo by degrading IFITM3.

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

Published: January 21, 2026

Lentiviral Gene Therapy for Severe Leukocyte Adhesion Deficiency Type 1.

Lentiviral Gene Therapy for Severe Leukocyte Adhesion Deficiency Type 1.

Journal: Obstetrical & gynecological survey

Published: December 30, 2025

DNA-PKcs inhibitor AZD7648 reveals sgRNA cross-contaminants and enhanced sensitivity of genome engineering off-target activity in HSPCs.

DNA-PKcs inhibitor AZD7648 reveals sgRNA cross-contaminants and enhanced sensitivity of genome engineering off-target activity in HSPCs.

Journal: Nucleic acids research

Published: December 18, 2025

Functional restoration of immune defects in STAT1 gain-of-function disease following stem cell gene editing.

Functional restoration of immune defects in STAT1 gain-of-function disease following stem cell gene editing.

Journal: Blood

Published: October 28, 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

In-depth characterization of stem cell potency and genotoxicity for clinical-scale ex vivo CRISPR-Cas9 gene editing.

In-depth characterization of stem cell potency and genotoxicity for clinical-scale ex vivo CRISPR-Cas9 gene editing.

Journal: Molecular therapy. Advances

Published: August 01, 2025

Lentiviral Gene Therapy for Severe Leukocyte Adhesion Deficiency Type 1.

Lentiviral Gene Therapy for Severe Leukocyte Adhesion Deficiency Type 1.

Journal: The New England journal of medicine

Published: April 30, 2025

Long-term efficacy and safety results of betibeglogene autotemcel gene therapy for transfusion-dependent β-thalassemia.

Long-term efficacy and safety results of betibeglogene autotemcel gene therapy for transfusion-dependent β-thalassemia.

Journal: Blood

Published: March 19, 2025

Long-term efficacy and safety results of betibeglogene autotemcel gene therapy for transfusion-dependent β-thalassemia.

Long-term efficacy and safety results of betibeglogene autotemcel gene therapy for transfusion-dependent β-thalassemia.

Journal: Blood

Published: March 19, 2025

Ex vivo modification of hematopoietic stem and progenitor cells for gene therapy.

Ex vivo modification of hematopoietic stem and progenitor cells for gene therapy.

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

Published: February 20, 2025

Preclinical evaluation of lentiviral gene therapy for adenosine deaminase 2 deficiency (DADA2): engraftment efficiency and biodistribution in humanised NBSGW mice.

Preclinical evaluation of lentiviral gene therapy for adenosine deaminase 2 deficiency (DADA2): engraftment efficiency and biodistribution in humanised NBSGW mice.

Journal: Gene therapy

Published: January 20, 2025

Showing 1-12 of 244

Last Updated: 09/26/2026