![Frequency and phenotypic characteristics of RPE65 mutations in the Chinese population | Orphanet Journal of Rare Diseases | Full Text Frequency and phenotypic characteristics of RPE65 mutations in the Chinese population | Orphanet Journal of Rare Diseases | Full Text](https://media.springernature.com/lw685/springer-static/image/art%3A10.1186%2Fs13023-021-01807-3/MediaObjects/13023_2021_1807_Fig1_HTML.png)
Frequency and phenotypic characteristics of RPE65 mutations in the Chinese population | Orphanet Journal of Rare Diseases | Full Text
![Mutations in the RPE65 gene in patients with autosomal recessive retinitis pigmentosa or Leber congenital amaurosis | PNAS Mutations in the RPE65 gene in patients with autosomal recessive retinitis pigmentosa or Leber congenital amaurosis | PNAS](https://www.pnas.org/cms/10.1073/pnas.95.6.3088/asset/9368dbcb-d7ab-4c48-9045-0358d7c95b10/assets/graphic/pq0584475003.jpeg)
Mutations in the RPE65 gene in patients with autosomal recessive retinitis pigmentosa or Leber congenital amaurosis | PNAS
![Lentiviral mediated RPE65 gene transfer in healthy hiPSCs-derived retinal pigment epithelial cells markedly increased RPE65 mRNA, but modestly protein level | Scientific Reports Lentiviral mediated RPE65 gene transfer in healthy hiPSCs-derived retinal pigment epithelial cells markedly increased RPE65 mRNA, but modestly protein level | Scientific Reports](https://media.springernature.com/full/springer-static/image/art%3A10.1038%2Fs41598-020-65657-y/MediaObjects/41598_2020_65657_Fig1_HTML.png)
Lentiviral mediated RPE65 gene transfer in healthy hiPSCs-derived retinal pigment epithelial cells markedly increased RPE65 mRNA, but modestly protein level | Scientific Reports
![IJMS | Free Full-Text | An Update on Gene Therapy for Inherited Retinal Dystrophy: Experience in Leber Congenital Amaurosis Clinical Trials IJMS | Free Full-Text | An Update on Gene Therapy for Inherited Retinal Dystrophy: Experience in Leber Congenital Amaurosis Clinical Trials](https://www.mdpi.com/ijms/ijms-22-04534/article_deploy/html/images/ijms-22-04534-g001-550.jpg)
IJMS | Free Full-Text | An Update on Gene Therapy for Inherited Retinal Dystrophy: Experience in Leber Congenital Amaurosis Clinical Trials
![Gene Therapy Restores Vision-Dependent Behavior as Well as Retinal Structure and Function in a Mouse Model of RPE65 Leber Congenital Amaurosis: Molecular Therapy Gene Therapy Restores Vision-Dependent Behavior as Well as Retinal Structure and Function in a Mouse Model of RPE65 Leber Congenital Amaurosis: Molecular Therapy](https://www.cell.com/cms/attachment/08ea434e-86e3-42c7-8e9c-0ded52291e8b/gr1.jpg)
Gene Therapy Restores Vision-Dependent Behavior as Well as Retinal Structure and Function in a Mouse Model of RPE65 Leber Congenital Amaurosis: Molecular Therapy
![RPE65 mutation frequency and phenotypic variation according to exome sequencing in a tertiary centre for genetic eye diseases in China - Li - 2020 - Acta Ophthalmologica - Wiley Online Library RPE65 mutation frequency and phenotypic variation according to exome sequencing in a tertiary centre for genetic eye diseases in China - Li - 2020 - Acta Ophthalmologica - Wiley Online Library](https://onlinelibrary.wiley.com/cms/asset/551f4ba9-d79d-4d28-8e29-d91211bf6adb/aos14181-fig-0003-m.jpg)
RPE65 mutation frequency and phenotypic variation according to exome sequencing in a tertiary centre for genetic eye diseases in China - Li - 2020 - Acta Ophthalmologica - Wiley Online Library
![Cells | Free Full-Text | The Predicted Splicing Variant c.11+5G>A in RPE65 Leads to a Reduction in mRNA Expression in a Cell-Specific Manner Cells | Free Full-Text | The Predicted Splicing Variant c.11+5G>A in RPE65 Leads to a Reduction in mRNA Expression in a Cell-Specific Manner](https://pub.mdpi-res.com/cells/cells-11-03640/article_deploy/html/images/cells-11-03640-ag.png?1669078265)