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Improved visual outcomes and quality of life GENE X GENE M cell NR2E3 Delivery “Molecular reset” of health & survival gene networks cell Cell with normal function Restored retinal cell homeostasis ONL ONL/photoreceptor survival GENE X Cell with mutated/ nonfunctioning gene(s) other than modifier gene MODIFIER GENE M GENE M cell 2
• • • Nuclear Hormone Receptors (NHRs): intracellular receptors that regulate gene expression, acting as a master regulator of genes in the retina.
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