1. Spark Therapeutics. FDA Briefing Document—Spark Therapeutics, Inc, LUXTURNATM: Cellular, Tissue, and Gene Therapies Advisory Committee Meeting, October 12, 2017. https://www.fda.gov/downloads/advisorycommittees/committeesmeetingmaterials/bloodvaccinesandotherbiologics/cellulartissueandgenetherapiesadvisorycommittee/ucm579300.pdf (accessed March 12, 2018).
2. International Council of Ophthalmology. Visual standards aspects and ranges of vision loss with emphasis on population surveys. 29th International Congress of Ophthalmology, Sydney, Australia, 2002.
3. Comprehensive genotyping reveals RPE65 as the most frequently mutated gene in Leber congenital amaurosis in Denmark;Astuti;Eur J Hum Genet,2016
4. Leber congenital amaurosis/early-onset severe retinal dystrophy: clinical features, molecular genetics and therapeutic interventions;Kumaran;Br J Ophthalmol,2017
5. The retinal pigment epithelium in health and disease;Sparrow;Curr Mol Med,2010