Publications by authors named "Ian Alexander"

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Targeted editing of the 21-hydroxylase locus confers durable therapeutic effect in a murine model of congenital adrenal hyperplasia.
Lara E Graves, Lakshmy Viswanath, Eva B van Dijk, Erhua Zhu, Sundar Koyyalamudi, Ian E Alexander

Mol Ther· November 2025

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Pancreatic transdifferentiation of NOD mouse livers prevented development of hyperglycemia.
Alexandra L G Mahoney, Binhai Ren, Najah T Nassif, Bronwyn A O'Brien, Grant J Logan, Ian E Alexander

Mol Ther· October 2025

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Gene therapy enhances deoxyribonuclease I treatment in antimyeloperoxidase glomerulonephritis.
Anne Cao Le, Virginie Oudin, Jonathan Dick, Maliha A Alikhan, Timothy A Gottschalk, Ian E Alexander

JCI Insight· August 2025

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High-throughput evaluation of cardiac-specific promoters for adeno-associated virus mediated cardiac gene therapy.
Dhanya Ravindran, Renuka Rao, Juan Mundisugih, Tracy Titus, Shinya Tsurusaki, Ian E Alexander

Gene Ther· July 2025

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Hepatitis C Treatment Initiation During Hospitalization for People Who Use Drugs: A Narrative Review of the Literature.
Jacob Denkins, Jane Babiarz, YoungYoon Ham, HaYoung Ryu, Brian Ricci, Ian Alexander

Open Forum Infect Dis· June 2025

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Structural characterization of antibody-responses following Zolgensma treatment for AAV capsid engineering to expand patient cohorts.
Mario Mietzsch, Jane Hsi, Austin R Nelson, Neeta Khandekar, Ann-Maree Huang, Ian E Alexander

Nat Commun· April 2025

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AAVolve: Concatenated long-read deep sequencing enables whole capsid tracking during shuffled AAV library selection.
Suzanne Scott, Adrian Westhaus, Deborah Nazareth, Marti Cabanes-Creus, Renina Gale Navarro, Ian E Alexander

Mol Ther Methods Clin Dev· December 2024

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Sleeping Beauty mRNA-LNP enables stable rAAV transgene expression in mouse and NHP hepatocytes and improves vector potency.
Philip M Zakas, Sharon C Cunningham, Ann Doherty, Eva B van Dijk, Raed Ibraheim, Ian E Alexander

Mol Ther· October 2024

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Gene therapy clinical trials worldwide to 2023-an update.
Samantha L Ginn, Mawj Mandwie, Ian E Alexander, Michael Edelstein, Mohammad R Abedi

J Gene Med· August 2024

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Stable transduction of the neonatal mouse liver using a hybrid rAAV/sleeping beauty transposon gene delivery system.
Sharon C Cunningham, Philip M Zakas, Natsuki Sasaki, Eva B van Dijk, Erhua Zhu, Ian E Alexander

J Gene Med· August 2024

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AAV-delivered hepato-adrenal cooperativity in steroidogenesis: Implications for gene therapy for congenital adrenal hyperplasia.
Lara E Graves, Eva B van Dijk, Erhua Zhu, Sundar Koyyalamudi, Tiffany Wotton, Ian E Alexander

Mol Ther Methods Clin Dev· June 2024

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Novel AAV variants with improved tropism for human Schwann cells.
Matthieu Drouyer, Tak-Ho Chu, Elodie Labit, Florencia Haase, Renina Gale Navarro, Ian E Alexander

Mol Ther Methods Clin Dev· June 2024

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Structural characterization of antibody-responses from Zolgensma treatment provides the blueprint for the engineering of an AAV capsid suitable for redosing.
Mario Mietzsch, Austin R Nelson, Jane Hsi, Jon Zachary, Lindsay Potts, Ian E Alexander

bioRxiv· May 2024

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Development of CNS tropic AAV1-like variants with reduced liver-targeting following systemic administration in mice.
Matthieu Drouyer, Jessica Merjane, Deborah Nazareth, Maddison Knight, Suzanne Scott, Ian E Alexander

Mol Ther· March 2024

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Harnessing whole human liver ex situ normothermic perfusion for preclinical AAV vector evaluation.
Marti Cabanes-Creus, Sophia H Y Liao, Renina Gale Navarro, Maddison Knight, Deborah Nazareth, Ian E Alexander

Nat Commun· March 2024

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Liver-specific adiponectin gene therapy suppresses microglial NLRP3-inflammasome activation for treating Alzheimer's disease.
Roy Chun-Laam Ng, Min Jian, Oscar Ka-Fai Ma, Ariya Weiman Xiang, Myriam Bunting, Ian E Alexander

J Neuroinflammation· March 2024

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Gene therapy for urea cycle defects: An update from historical perspectives to future prospects.
Claire Duff, Ian E Alexander, Julien Baruteau

J Inherit Metab Dis· January 2024

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Recapitulation of Skewed X-Inactivation in Female Ornithine Transcarbamylase-Deficient Primary Human Hepatocytes in the FRG Mouse: A Novel System for Developing Epigenetic Therapies.
Sharon C Cunningham, Eva B van Dijk, Erhua Zhu, Maya Sugden, Mawj Mandwie, Ian E Alexander

Hum Gene Ther· September 2023

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Development of new adeno-associated virus capsid variants for targeted gene delivery to human cardiomyocytes.
Cindy Y Kok, Shinya Tsurusaki, Marti Cabanes-Creus, Sindhu Igoor, Renuka Rao, Ian E Alexander

Mol Ther Methods Clin Dev· September 2023

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Gene Therapy for Paediatric Homozygous Familial Hypercholesterolaemia.
Lara E Graves, Ari Horton, Ian E Alexander, Shubha Srinivasan

Heart Lung Circ· July 2023

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Structural and functional characterization of capsid binding by anti-AAV9 monoclonal antibodies from infants after SMA gene therapy.
Grant J Logan, Mario Mietzsch, Neeta Khandekar, Arlene D'Silva, Daniel Anderson, Ian E Alexander

Mol Ther· July 2023

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In Search of Adeno-Associated Virus Vectors With Enhanced Cardiac Tropism for Gene Therapy.
Natsuki Sasaki, Cindy Y Kok, Adrian Westhaus, Ian E Alexander, Leszek Lisowski

Heart Lung Circ· July 2023

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Future Directions for Adrenal Insufficiency: Cellular Transplantation and Genetic Therapies.
Lara E Graves, David J Torpy, P Toby Coates, Ian E Alexander, Stefan R Bornstein

J Clin Endocrinol Metab· May 2023

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Assessment of Pre-Clinical Liver Models Based on Their Ability to Predict the Liver-Tropism of Adeno-Associated Virus Vectors.
Adrian Westhaus, Marti Cabanes-Creus, Kimberley L Dilworth, Erhua Zhu, David Salas Gómez, Ian E Alexander

Hum Gene Ther· April 2023

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Characterization of the humanized FRG mouse model and development of an AAV-LK03 variant with improved liver lobular biodistribution.
Marti Cabanes-Creus, Renina Gale Navarro, Sophia H Y Liao, Suzanne Scott, Rodrigo Carlessi, Ian E Alexander

Mol Ther Methods Clin Dev· March 2023

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