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Review article

First published 2015-2

Adeno-Associated Virus Vectors and Neurological Gene Therapy

David S. Ojala, Dominic P. Amara, David V. Schaffer

The Neuroscientist

Free access

Research article

First published 2018-12

Considerations for Preclinical Safety Assessment of Adeno-Associated Virus Gene Therapy Products

Basel T. Assaf, Laurence O. Whiteley

Toxicologic Pathology

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Article commentary

First published 2025-11

The Responsible Development of Adeno-Associated Virus Gene Therapies

James M. Wilson, Arthur L. Caplan

Human Gene Therapy

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Review article

First published 2016-12

Adeno-Associated Virus Gene Therapy for Liver Disease

Lisa M. Kattenhorn, Christopher H. Tipper, Lorelei Stoica [...]

Human Gene Therapy

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Review article

First published 2016-07

Adeno-Associated Virus-Based Gene Therapy for CNS Diseases

Michaël Hocquemiller, Laura Giersch, Mickael Audrain [...]

Human Gene Therapy

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Research article

First published 2020-05

Gene Therapy for Cystic Fibrosis Paved the Way for the Use of Adeno-Associated Virus in Gene Therapy

William B. Guggino, Liudmila Cebotaru

Human Gene Therapy

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Review article

First published 2013-11

Adeno-Associated Virus–Mediated Gene Therapy for Metabolic Myopathy

Cathryn S. Mah, Meghan S. Soustek, A. Gary Todd [...]

Human Gene Therapy

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Research article

First published 2022-09

Redosing Adeno-Associated Virus Gene Therapy to the Central Nervous System

Abigail McElroy, Miguel Sena-Esteves, Motahareh Arjomandnejad [...]

Human Gene Therapy

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Research article

First published 2004-02

Recombinant Adeno-Associated Virus As Delivery Vector for Gene Therapy—A Review

Yang Lu

Stem Cells and Development

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Review article

First published 2018-12

Translational Aspects of Adeno-Associated Virus–Mediated Cardiac Gene Therapy

Tilman Ziegler, Kiyotake Ishikawa, Rabea Hinkel [...]

Human Gene Therapy

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