Gene expression profile from single cell level of retinal cells from optic cup of injected Abca4KO mice with miR-181a/b sponge therapeutic

We observed a beneficial effect on Abca4KO mice phenotype upon miR-181a/b sponge treatment. Then, we decided to perform single cell analysis to better understand the pathway associated with amelioration observed. Abca4KO mice were injected with miR-181a/b sponge in one eye and with a control vector in the controlateral. Subretinal injection were performed at 2 months of age and samples collected and analyzed at 4 months post-treatment. In a second experiment Abca4KO and WT mice were analyzed analyzed at 4 months.