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. 2022 Aug 24;29:823–835. doi: 10.1016/j.omtn.2022.08.026

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© 2022 The Author(s)

This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/).

PMC Copyright notice

Gene expression and therapeutic efficacy of repeated dosing of PEG-ECO/pGRK1-ABCA4-S/MAR nanoparticles in Abca4^−/− mice

(A) Timeline of repeated injections of PEG-ECO/pGRK1-ABCA4-S/MAR nanoparticles. (B) Time course of ABCA4 mRNA expression, analyzed by qRT-PCR after 1 injection of PEG-ECO/pGRK1-ABCA4-S/MAR nanoparticles (100 ng/eye) in Abca4^−/− mice. (C and D) Fluorescence IHC staining of ABCA4 (green) and RPE65 (red) of retina sections from Abca4^−/− mice that received 1, 2, and 3 subretinal injections of PEG-ECO/pGRK1-ABCA4-S/MAR (100 ng/eye; blue, DAPI) (C) and of the whole retina after 3 injections (D). (E) qRT-PCR of ABCA4 mRNA expression in Abca4^−/− mice that received 1, 2, and 3 injections of PEG-ECO/pGRK1-ABCA4-S/MAR. (F) Representative HPLC chromatograms of A2E in the eyes of Abca4^−/− mice treated with PEG-ECO/pGRK1-ABCA4-S/MAR nanoparticles in comparison with that in control mice. (G) Quantitative comparison of A2E levels in treated Abca4^−/− mice relative to control mice. ∗p < 0.05, ∗∗p < 0.01, ∗∗∗p < 0.005, ∗∗∗∗p < 0.001.