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. 2024 Mar 19;16(4):805–822. doi: 10.1038/s44321-024-00053-x

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© The Author(s) 2024

Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article’s Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article’s Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/. Creative Commons Public Domain Dedication waiver http://creativecommons.org/publicdomain/zero/1.0/ applies to the data associated with this article, unless otherwise stated in a credit line to the data, but does not extend to the graphical or creative elements of illustrations, charts, or figures. This waiver removes legal barriers to the re-use and mining of research data. According to standard scholarly practice, it is recommended to provide appropriate citation and attribution whenever technically possible.

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(A) Retinal activity recorded by ERG in scotopic conditions 3 mpi in Fam161a^tm1b/tm1b mice of either IRBP-GRK1-HS or HL vectors (10¹⁰ GC) at PN14 and in the contralateral eye with IRBP-GRK1-GFP (untreated: n = 4, GFP: n = 9, HL: n = 6, HS: n = 7, all at a dose of 10¹⁰ GC/injection) showed no functional improvement in the hFAM161A-treated eyes. (B–D) The retinal activity was determined in Fam161a^tm1b/tm1b mice injected at PN14 with a mix of the two vectors (HS + HL) and monitored by ERG after 1 month (B, n = 18), 2 months (C, n = 18) and 3 months (D, n = 12). Data are expressed as mean + SEM. No improvement was observed at any of these time points. Expression pattern of FAM161A in WT (E), KO (F), and 6 mpi after injection with IRBP-GRK1-HS (10¹⁰ GC, G) or –HL (H), or 3 mpi after injection with IRBP-GRK1-HS + IRBP-GRK1-HL (I) into Fam161a^tm1b/tm1b mouse eyes. Endogenous FAM161A expression is pointed by white arrows in (E). Note the ectopic expression of FAM161A (orange arrows, G–I). In addition, the CC is not distinguishable. (J–M) ONL thickness 6 months post injection of either HS or HL vectors. Note the thicker ONL in FAM161A-positive areas compared to FAM161A-negative region. (N) The combination of HS and HL isoforms (n = 9, 10¹⁰ GC in total) enhanced retina thickness rescue in comparison to the non-treated area (n = 9, P < 0.001) to reach the WT (n = 5) retina thickness. Scales bars are 25 μm. ONL outer nuclear layer, HS human short isoform, HL human long isoform. A one-way ANOVA Kruskal–Wallis test was performed, followed by Dunn’s test for multiple comparisons between the groups. *P < 0.05; ***P < 0.001. Data information: The result in (E) is representative of immunolabeling performed on two eyes from two individual mice. The result in (F) is representative of three eyes from three individual mice as well as of the non-treated regions of all AAV-treated eyes of the experiments analyzed in this study. Results in (G–M) are representative of five independent experiments with the respective number of eyes: HS: n = 18, HL: n = 18, and HS + HL: n = 12. Source data are available online for this figure.