Subject Areas on Research
- A new era in medical therapy for retinal degenerative disease?
- Comprehensive molecular diagnosis of 179 Leber congenital amaurosis and juvenile retinitis pigmentosa patients by targeted next generation sequencing.
- Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years.
- Knocking out lca5 in zebrafish causes cone-rod dystrophy due to impaired outer segment protein trafficking.