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RPE65: role in the visual cycle, human retinal disease, and gene therapy.
Persistence of non-viral vector mediated RPE65 expression: case for viability as a gene transfer therapy for RPE-based diseases.
S/MAR-containing DNA nanoparticles promote persistent RPE gene expression and improvement in RPE65-associated LCA.
Episomal maintenance of S/MAR-containing non-viral vectors for RPE-based diseases.
cis trans Isomerases