Article

Efficacy and Safety of Intravitreal rAAV2-ND4 Therapy for Leber’s Hereditary Optic Neuropathy

2024-11-27

Abstract excerpt

<title>Abstract</title> <p>Background: This study aimed to assess the safety and efficacy of a rAAV2 carrying normal <italic>ND4</italic> (rAAV2-ND4) (NR082) in individuals with visual loss due to LHON carrying the m.11778G > A mutation. Additionally, it aimed to determine a safe dose of NR082 for intravitreal injection. <h4>Methods:</h4> This is a single-arm, open-label, dose-finding clinical trial. A total of...

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Literature Corpus work: 48b22bc0-51fe-5307-bb9b-fc204c927a21
DOI: 10.21203/rs.3.rs-5307802/v1

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