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1-2 of 2 results for AAV9 vector-based gene replacement therapy

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Select article: Knockdown-Replace: A New Dynamic for Treatment of Dynamin-1 Related developmental and epileptic encephalopathy (DEE)

Open access

Article commentary

First published December, 2025

Knockdown-Replace: A New Dynamic for Treatment of Dynamin-1 Related developmental and epileptic encephalopathy (DEE)

Jacy L. Wagnon

Epilepsy Currents

Select article: Real-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy: Findings from the RESTORE Registry

Open access

Research article

First published March 5, 2024

Real-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy: Findings from the RESTORE Registry

Laurent Servais, John W. Day, Darryl C. De Vivo [...]

Journal of Neuromuscular Diseases

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