Gegnaw Shumet T, Sandu Cristina, Bery Amandine, Ten Brink Jacoline B, Milićević Nemanja, Jongejan Aldo, Moerland Perry D, Bergen Arthur A, Felder-Schmittbuhl Marie-Paule
Centre National de la Recherche Scientifique, Université de Strasbourg, Institut Des Neurosciences Cellulaires et Intégratives, Strasbourg, France.
Department of Human Genetics, Amsterdam University Medical Centers, Location AMC, University of Amsterdam, Amsterdam, The Netherlands.
FASEB J. 2025 Apr 15;39(7):e70507. doi: 10.1096/fj.202401967R.
Daily rhythms are a central hallmark of vision, in particular by adapting retinal physiology and light response to the day-night cycle. These cyclic processes are regulated by retinal circadian clocks, molecular machineries regulating gene expression across the 24-h cycle. Although hundreds of genes associated with genetic retinal disorders have been identified, no direct link has been established with the clock. Hence, we investigated the hypothesis that a poorly functioning circadian clock aggravates retinal photoreceptor disease. We performed this study in the P23H rhodopsin-mutated mouse model (P23H Rho) that mimics one major cause of human autosomal dominant retinitis pigmentosa. We also used the rod-specific knockout (rod-Bmal1KO) of Bmal1, a key clock component. More specifically, we used either heterozygous P23H Rho mice or rod-Bmal1KO alone, as well as double mutants of these strains and control mice. We showed by structural (histology, immunohistochemistry) and functional (electroretinography: ERG) analyses that the retinitis pigmentosa phenotype is exacerbated in the double mutant line compared to the P23H Rho mutation alone. Indeed, we observed marked ERG amplitude reduction and more photoreceptor cell loss in double mutants with respect to simple P23H Rho mutants. These observations were further corroborated by transcriptome analysis revealing major gene expression differences between these genotypes. In this data, we identified unique gene expression sets implicating neurogenesis, phototransduction cascade, and metabolism, associated with enhanced photoreceptor degeneration. Thus, our results establish a link between clock dysfunction and retinal degeneration and suggest underlying molecular mechanisms, together providing new concepts for understanding and managing blinding diseases.
日常节律是视觉的一个核心特征,特别是通过使视网膜生理和光反应适应昼夜循环来体现。这些周期性过程由视网膜生物钟调节,生物钟是在24小时周期内调节基因表达的分子机制。尽管已经鉴定出数百个与遗传性视网膜疾病相关的基因,但尚未建立与生物钟的直接联系。因此,我们研究了生物钟功能不良会加重视网膜光感受器疾病这一假说。我们在模拟人类常染色体显性视网膜色素变性主要病因之一的P23H视紫红质突变小鼠模型(P23H Rho)中进行了这项研究。我们还使用了生物钟关键成分Bmal1的视杆细胞特异性敲除小鼠(视杆细胞-Bmal1KO)。更具体地说,我们使用了杂合的P23H Rho小鼠或单独的视杆细胞-Bmal1KO,以及这些品系的双突变体和对照小鼠。我们通过结构分析(组织学、免疫组织化学)和功能分析(视网膜电图:ERG)表明,与单独的P23H Rho突变相比,双突变系中的视网膜色素变性表型更加严重。事实上,我们观察到双突变体相对于单纯的P23H Rho突变体,ERG振幅明显降低,光感受器细胞损失更多。转录组分析揭示了这些基因型之间的主要基因表达差异,进一步证实了这些观察结果。在这些数据中,我们确定了与神经发生、光转导级联和代谢相关的独特基因表达集,这些基因表达集与光感受器变性增强有关。因此,我们的结果建立了生物钟功能障碍与视网膜变性之间的联系,并提出了潜在的分子机制,共同为理解和治疗致盲疾病提供了新的概念。