Using patient-specific induced pluripotent stem cells to interrogate the pathogenicity of a novel retinal pigment epithelium-specific 65Â kDa cryptic splice site mutation and confirm eligibility for enrollment into a clinical gene augmentation trial
Keywords: اپیتلیوم رنگدانه شبکیه; Best1; Bestrophin1; bFGF; basic fibroblast growth factor; DMEM; Dulbecco's Modified Eagle Medium; DNMT; DNA methyltransferases; ERG; Electroretinography; KLF4; Kruppel-like factor 4; LB; Lysogeny broth; LCA; Leber congenital amaurosis; MEM; minimum essent