Ian E. Alexander

Westmead, NSW 2145, Australia

Last Updated: 09/26/2026

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Found 105 publications

Functional Editing of the OTC locus by Targeted Integration with Phenotype Correction and Restoration of Physiological Patterns of Expression.

Functional Editing of the OTC locus by Targeted Integration with Phenotype Correction and Restoration of Physiological Patterns of Expression.

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

Published: January 28, 2026

Identification of AAV Vector Integration Sites in Primary Human and Macaque Hepatocytes in vivo, and Analysis of Oncogenic Risk.

Identification of AAV Vector Integration Sites in Primary Human and Macaque Hepatocytes in vivo, and Analysis of Oncogenic Risk.

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

Published: January 20, 2026

A novel fusion protein reduces kidney complement in experimental C3 glomerulopathy.

A novel fusion protein reduces kidney complement in experimental C3 glomerulopathy.

Journal: Clinical and experimental immunology

Published: December 05, 2025

Exploration of the potential of genomic editing in the treatment of congenital adrenal hyperplasia.

Exploration of the potential of genomic editing in the treatment of congenital adrenal hyperplasia.

Journal: Frontiers in endocrinology

Published: October 06, 2025

Targeted editing of the 21-hydroxylase locus confers durable therapeutic effect in a murine model of congenital adrenal hyperplasia.

Targeted editing of the 21-hydroxylase locus confers durable therapeutic effect in a murine model of congenital adrenal hyperplasia.

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

Published: June 05, 2025

High-throughput evaluation of cardiac-specific promoters for adeno-associated virus mediated cardiac gene therapy.

High-throughput evaluation of cardiac-specific promoters for adeno-associated virus mediated cardiac gene therapy.

Journal: Gene therapy

Published: February 23, 2025

Pancreatic transdifferentiation of NOD mouse livers prevented development of hyperglycemia.

Pancreatic transdifferentiation of NOD mouse livers prevented development of hyperglycemia.

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

Published: January 01, 2025

Gene therapy enhances deoxyribonuclease I treatment in antimyeloperoxidase glomerulonephritis.

Gene therapy enhances deoxyribonuclease I treatment in antimyeloperoxidase glomerulonephritis.

Journal: JCI insight

Published: November 07, 2024

AAVolve: Concatenated long-read deep sequencing enables whole capsid tracking during shuffled AAV library selection.

AAVolve: Concatenated long-read deep sequencing enables whole capsid tracking during shuffled AAV library selection.

Journal: Molecular therapy. Methods & clinical development

Published: July 09, 2024

Structural characterization of antibody-responses from Zolgensma treatment provides the blueprint for the engineering of an AAV capsid suitable for redosing.

Structural characterization of antibody-responses from Zolgensma treatment provides the blueprint for the engineering of an AAV capsid suitable for redosing.

Journal: bioRxiv : the preprint server for biology

Published: May 15, 2024

Structural characterization of antibody-responses following Zolgensma treatment for AAV capsid engineering to expand patient cohorts.

Structural characterization of antibody-responses following Zolgensma treatment for AAV capsid engineering to expand patient cohorts.

Journal: Nature communications

Published: April 15, 2024

Gene therapy clinical trials worldwide to 2023-an update.

Gene therapy clinical trials worldwide to 2023-an update.

Journal: The journal of gene medicine

Published: March 12, 2024

Showing 1-12 of 105

Last Updated: 09/26/2026