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Open access

Research article

First published 2022-01-04

Pre Clinical Assessment of AAVrh74.MCK.GNE Viral Vector Therapeutic Potential: Robust Activity Despite Lack of Consistent Animal Model for GNE Myopathy

Stella Mitrani-Rosenbaum, Lena Yakovlev, Michal Becker Cohen [...]

Journal of Neuromuscular Diseases

Open access

Research article

First published 2025-11

Quantification and comparison of anti-AAV9 and anti-AAVrh74 antibodies in plasma and human milk: Implications for AAV-based gene therapy candidacy

Carmen Leon-Astudillo, Kirsten Coleman, Stephanie M Salabarria [...]

Journal of Neuromuscular Diseases

Retricted access

Research article

First published 2026-1

Effective Treatment of Colq-Deficient Mice with Adeno-Associated Virus Type Rh74-Mediated Gene Therapy

Abigail McInnes, Jaime N. Young, Anahid S. Aivazian [...]

Human Gene Therapy

Retricted access

Research article

First published 2023-05

Seroprevalence of Adeno-Associated Virus Neutralizing Antibodies in Males with Duchenne Muscular Dystrophy

Sumit Verma, Stella N. Nwosu, Raj Razdan [...]

Human Gene Therapy

Open access

Research article

First published 2023

Evaluation of rAAVrh74 gene therapy vector seroprevalence by measurement of total binding antibodies in patients with Duchenne muscular dystrophy

Natalie L. Goedeker, Sachi D. Dharia, Danielle A. Griffin [...]

Therapeutic Advances in Neurological Disorders

Retricted access

Research article

First published 2017-12

Differential Prevalence of Antibodies Against Adeno-Associated Virus in Healthy Children and Patients with Mucopolysaccharidosis III: Perspective for AAV-Mediated Gene Therapy

Haiyan Fu, Aaron S. Meadows, Ricardo J. Pineda [...]

Human Gene Therapy Clinical Development

Open access

Research article

First published 2026-3

Learnings from Patient Mortality after Delandistrogene Moxeparvovec Administration: A Report of Two Cases and Expert Committee Considerations for Future Mitigation and Management

Perry B. Shieh, Crystal Proud, Tamir Diamond [...]

Human Gene Therapy

Retricted access

Research article

First published 2019-07

Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D by Isolated Limb Infusion

Jerry R. Mendell, Louis G. Chicoine, Samiah A. Al-Zaidy [...]

Human Gene Therapy

Open access

Research article

First published 2021-04

Preclinical Systemic Delivery of Adeno-Associated α-Sarcoglycan Gene Transfer for Limb-Girdle Muscular Dystrophy

Danielle A. Griffin, Eric R. Pozsgai, Kristin N. Heller [...]

Human Gene Therapy

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