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Palabras clave – provistas por la editorial

induced pluripotent stem cell (iPSC); clustered regularly interspaced short palindromic repeats (CRISPR); homology-directed repair (HDR); Enhanced S-Cone Syndrome (ESCS); NR2E3; AAV; retina; gene therapy; dual AAV; gold nanoparticles; DNA-wrapped gold nanoparticles; ARPE-19 cells; retinal pigment epithelium; clathrin-coated vesicles; endosomal trafficking; retinitis pigmentosa; autosomal dominant; G56R; putative dominant negative effect; gapmer antisense oligonucleotides; allele-specific knockdown; Leber congenital amaurosis and allied retinal ciliopathies; CEP290; Flanders founder c.4723A > T nonsense mutation; Cilia elongation; spontaneous nonsense correction; AON-mediated exon skipping; microRNA; photoreceptors; rods; cones; bipolar cells; Müller glia; retinal inherited disorders; retinal degeneration; antisense oligonucleotides; Stargardt disease; inherited retinal diseases; splicing modulation; RNA therapy; ABCA4; iPSC-derived photoreceptor precursor cells; cyclic GMP; apoptosis; necrosis; drug delivery systems; translational medicine; Usher syndrome; Leber congenital amaurosis; RPE65; nonprofit; patient registry; translational; protein trafficking; protein folding; protein degradation; chaperones; chaperonins; heat shock response; unfolded protein response; autophagy; therapy; IRD; DNA therapies; RNA therapies; compound therapies; clinical trials; Retinitis Pigmentosa GTPase Regulator; adeno-associated viral; Retinitis Pigmentosa (RP); choroideremia; REP1; inherited retinal disease; treatment; apical polarity; crumbs complex; fetal retina; PAR complex; retinal organoids; retinogenesis; gene augmentation; adeno-associated virus (AAV); n/a