Publication | Open Access

Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial

Translational MedicineGene TherapiesOcular DiseaseAav2 Gene TherapyIn Vivo Gene TherapyOphthalmologyContralateral-eye AdministrationExperimental OphthalmologyRpe65 MutationsMedicine