Associated Papers
Gene Therapy with Voretigene Neparvovec Improves Vision and Partially Restores Electrophysiological Function in Pre-School Children with Leber Congenital Amaurosis.
Gerhardt MJ et al.
Biomedicines, 2022
Clinical Features and Natural History in a Cohort of Chinese Patients with RPE65-Associated Inherited Retinal Dystrophy.
Shi J et al.
Journal of Clinical Medicine, 2021
Compound heterozygous RPE65 mutations associated with an early onset autosomal recessive retinitis pigmentosa.
Owczarek-Lipska M et al.
The Journal of Gene Medicine, 2020
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