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双重遗传模式:一系列具有不同分子机制的非综合征性遗传性视网膜疾病表型。

Dual inheritance patterns: A spectrum of non-syndromic inherited retinal disease phenotypes with varying molecular mechanisms.

作者信息

Holtes Lara K, de Bruijn Suzanne E, Cremers Frans P M, Roosing Susanne

机构信息

Department of Human Genetics, Radboud University Medical Center, Nijmegen, the Netherlands.

Department of Human Genetics, Radboud University Medical Center, Nijmegen, the Netherlands.

出版信息

Prog Retin Eye Res. 2025 Jan;104:101308. doi: 10.1016/j.preteyeres.2024.101308. Epub 2024 Oct 31.

DOI:10.1016/j.preteyeres.2024.101308
PMID:39486507
Abstract

Inherited retinal diseases (IRDs) encompass a variety of disease phenotypes and are known to display both clinical and genetic heterogeneity. A further complexity is that for several IRD-associated genes, pathogenic variants have been reported to cause either autosomal dominant (AD) or autosomal recessive (AR) diseases. The possibility of dual inheritance can create a challenge for variant interpretation as well as the genetic counselling of patients. This review aims to determine whether the molecular mechanisms behind the dual inheritance of each IRD-associated gene is well established, not yet properly understood, or if the association is questionable. Each gene is discussed individually in detail due to different protein structures and functions, but there are overlapping characteristics. For example, eight genes only have a limited number of reported pathogenic variants or a hotspot region implicated in the second inheritance pattern. Whereas CRX and RP1 display distinct spatial patterns for AR and AD pathogenic variants based on the variant type and/or location. The genes with a questionable dual inheritance, namely AIPL1, CRB1, and RCBTB1 highlight the importance of carefully considering allele frequency data. Finally, the crucial role relevant functional studies in animal and cell models play in validating a variant's biochemical or molecular effect is emphasised.

摘要

遗传性视网膜疾病(IRD)包含多种疾病表型,已知具有临床和遗传异质性。另一个复杂之处在于,对于一些与IRD相关的基因,已报道致病变异可导致常染色体显性(AD)或常染色体隐性(AR)疾病。双重遗传的可能性会给变异解读以及患者的遗传咨询带来挑战。本综述旨在确定每个与IRD相关基因双重遗传背后的分子机制是否已得到充分确立、尚未得到充分理解,或者这种关联是否存在疑问。由于蛋白质结构和功能不同,每个基因都单独进行了详细讨论,但也有重叠的特征。例如,八个基因仅有数量有限的已报道致病变异,或者存在与第二种遗传模式相关的热点区域。而CRX和RP1根据变异类型和/或位置,在AR和AD致病变异方面表现出不同的空间模式。具有可疑双重遗传的基因,即AIPL1、CRB1和RCBTB1,突出了仔细考虑等位基因频率数据的重要性。最后,强调了动物和细胞模型中的相关功能研究在验证变异的生化或分子效应方面所起的关键作用。

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