1. Miraldi Utz, Virginia, Coussa, Razek Georges, Antaki, Fares, Traboulsi, Elias I. 2018. Gene therapy for RPE65-related retinal disease. In Ophthalmic genetics, 39, 671-677. doi:10.1080/13816810.2018.1533027. https://pubmed.ncbi.nlm.nih.gov/30335549/
2. Kahraman, Neslihan Sinim, Öner, Ayşe, Özkul, Yusuf, Dündar, Munis. . Frequency of RPE65 Gene Mutation in Patients with Hereditary Retinal Dystrophy. In Turkish journal of ophthalmology, 52, 270-275. doi:10.4274/tjo.galenos.2021.74944. https://pubmed.ncbi.nlm.nih.gov/36017377/
3. Wang, Xue, Yu, Chaofeng, Tzekov, Radouil T, Zhu, Yihua, Li, Wensheng. 2020. The effect of human gene therapy for RPE65-associated Leber's congenital amaurosis on visual function: a systematic review and meta-analysis. In Orphanet journal of rare diseases, 15, 49. doi:10.1186/s13023-020-1304-1. https://pubmed.ncbi.nlm.nih.gov/32059734/
4. Pierce, Eric A, Bennett, Jean. 2015. The Status of RPE65 Gene Therapy Trials: Safety and Efficacy. In Cold Spring Harbor perspectives in medicine, 5, a017285. doi:10.1101/cshperspect.a017285. https://pubmed.ncbi.nlm.nih.gov/25635059/
5. Sallum, Juliana M F, Kaur, Vinay Preet, Shaikh, Javed, Viriato, Daniel, Fischer, M Dominik. 2022. Epidemiology of Mutations in the 65-kDa Retinal Pigment Epithelium (RPE65) Gene-Mediated Inherited Retinal Dystrophies: A Systematic Literature Review. In Advances in therapy, 39, 1179-1198. doi:10.1007/s12325-021-02036-7. https://pubmed.ncbi.nlm.nih.gov/35098484/
6. Maguire, Albert M, Simonelli, Francesca, Pierce, Eric A, High, Katherine A, Bennett, Jean. 2008. Safety and efficacy of gene transfer for Leber's congenital amaurosis. In The New England journal of medicine, 358, 2240-8. doi:10.1056/NEJMoa0802315. https://pubmed.ncbi.nlm.nih.gov/18441370/
7. Stein, Linda, Roy, Kamolika, Lei, Lei, Kaushal, Shalesh. . Clinical gene therapy for the treatment of RPE65-associated Leber congenital amaurosis. In Expert opinion on biological therapy, 11, 429-39. doi:10.1517/14712598.2011.557358. https://pubmed.ncbi.nlm.nih.gov/21299439/
智鼠故事 
