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Research article

First published 2026-5

A Tripartite AAV System with Engineered Lox Sites Enables Efficient Delivery of the EYS Gene for Retinal Gene Therapy

Kun-Do Rhee, Poppy Datta, Clairissa Baccam [...]

Human Gene Therapy

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Research article

First published 2024-12

Pathology Findings and In-Life Correlates in the Nonclinical Development of Adeno-Associated Virus (AAV)-Based Retinal Gene Therapies

Helen S. Booler, Typhaine Lejeune, Oliver Turner [...]

Toxicologic Pathology

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Research article

First published 2024-12

Neuropathological Findings in Nonclinical Species Following Administration of Adeno-Associated Virus (AAV)-Based Gene Therapy Vectors

Brad Bolon, Elizabeth Buza, Elizabeth Galbreath [...]

Toxicologic Pathology

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Research article

First published 2007-02

Functions of AAV-CMV-F.IX And AAV-EF1α-F.IX in Gene Therapy for Hemophilia B

Viroj Wiwanitkit

Human Gene Therapy

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Meeting report

First published 2023-10

An Overview of Nonclinical and Clinical Liver Toxicity Associated With AAV Gene Therapy

Laurence O. Whiteley

Toxicologic Pathology

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Review article

First published 2024-05

Development of AAV-Mediated Gene Therapy Approaches to Treat Skeletal Diseases

Chujiao Lin, Matthew B. Greenblatt, Guangping Gao [...]

Human Gene Therapy

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Review article

First published 2013-04

Pre-existing Anti–Adeno-Associated Virus Antibodies as a Challenge in AAV Gene Therapy

Vedell Louis Jeune, Jakob A. Joergensen, Roger J. Hajjar [...]

Human Gene Therapy Methods (Part B)

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Review article

First published 2025-6

Mutagenesis and Carcinogenesis Risk Evaluation for AAV and Lentiviral Gene Therapies

Toufan Parman, Daniella M. Pizzurro, Jacquelynn Lucas [...]

International Journal of Toxicology

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Research article

First published 2018-03

Dual AAV Gene Therapy for Duchenne Muscular Dystrophy with a 7-kb Mini-Dystrophin Gene in the Canine Model

Kasun Kodippili, Chady H. Hakim, Xiufang Pan [...]

Human Gene Therapy

Open access

Review article

First published 2026

Translational insights from nonclinical studies of AAV gene therapies for hemophilia: mechanisms underpinning variability and durability of gene expression

Sylvia Fong, Laura L. Swystun, Paul Batty [...]

Therapeutic Advances in Hematology

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