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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

JAMA Ophthalmology Feb 13, 2019

Bouquet C, et al. - In this secondary analysis of a clinical trial, 15 candidates were studied to estimate the relation of the immune response after intravitreal injection of recombinant adeno-associated virus 2 carrying the ND4 gene in subjects with Leber hereditary optic neuropathy (LHON) with intraocular inflammation. They noticed 13 individuals experiencing the intraocular inflammation after rAAV2/2-ND4 administration. The humoral immune response was not possibly correlated with the dose administered or with the immune status at baseline. They found no relationship between the ocular inflammation score (OIS) and the serum neutralizing antibody (NAb) titers. Overall, they suggested intravitreal administration of rAAV2/2-ND4 in subjects with LHON, safe and well tolerated.
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