Immune Response and Intraocular Inflammation in Patients With Leber Hereditary Optic Neuropathy Treated With Intravitreal Injection of Recombinant Adeno-Associated Virus 2 Carrying the ND4 Gene: A Secondary Analysis of a Phase 1/2 Clinical Trial.
Bouquet Céline, Vignal Clermont Catherine, Galy Anne, Fitoussi Serge, Blouin Laure, Munk Marion R et al.
This study found that intravitreal gene therapy for LHON caused mild, treatable intraocular inflammation, largely unrelated to viral dose or systemic immune response.