Our lab developed two modifier therapies using the nuclear hormone receptors (NHRs), NR2E3 and RORA, and the clinical trials for these therapies are being spearheaded by Ocugen Inc. A phase I/II clinical trial using an AAV to deliver NR2E3 subretinally in patients with RHO- and NR2E3-associated RP and CEP290-associated LCA has demonstrated both the safety and efficacy of NR2E3 modifier gene therapy in multiple diseases [9] (NCT05203939). Here, CEP290 is linked to Leber congenital amaurosis.