Over the last decades, significant progress has been made in ocular gene therapy, culminating in the approval of Voretigene neparvovec, the first gene therapy for retinal dystrophies caused by biallelic mutations in the RPE65 gene, such as Leber congenital amaurosis type 2. This chapter provides an overview of the gene therapy Voretigene neparvovec explaining the therapy's structure, mechanism of action, and the surgical procedure involved. Furthermore, it highlights important considerations for planning, executing, and following up on the therapy.