Safety of Lenadogene Nolparvovec Gene Therapy Over 5 Years in 189 Patients With Leber Hereditary Optic Neuropathy.

Catherine Vignal-Clermont,Patrick Yu-Wai-Man,Nancy J Newman,Valerio Carelli,Mark L Moster,Valerie Biousse,Prem S Subramanian,An-Guor Wang,Sean P Donahue,Bart P Leroy,Alfredo A Sadun,Thomas Klopstock,Robert C Sergott, Gema Rebolleda Fernandez,Bart K Chwalisz,Rudrani Banik,Magali Taiel,Michel Roux,José-Alain Sahel

American journal of ophthalmology（2023）

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摘要

Lenadogene nolparvovec had a good overall safety profile with excellent systemic tolerability, consistent with limited bio-dissemination. The product was well tolerated, with mostly mild ocular side effects responsive to conventional ophthalmologic treatments.

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关键词

Intravitreal gene therapy,Leber hereditary optic neuropathy,Safety

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