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RetiGene,一本用于遗传性视网膜疾病的综合基因图谱。

RetiGene, a comprehensive gene atlas for inherited retinal diseases.

作者信息

Rivolta Carlo, Celik Elifnaz, Kamdar Dhryata, Cancellieri Francesca, Kaminska Karolina, Ullah Mukhtar, Barberán-Martínez Pilar, Bouckaert Manon, Cortón Marta, Delanote Emma, Fernández-Caballero Lidia, García García Gema, Holtes Lara K, Karali Marianthi, Lopez Irma, Peter Virginie G, Schneider Nina, Vincke Lieselot, Ayuso Carmen, Banfi Sandro, Bocquet Beatrice, Coppieters Frauke, Cremers Frans P M, Inglehearn Chris F, Iwata Takeshi, Kalatzis Vasiliki, Koenekoop Robert K, Millán José M, Sharon Dror, Toomes Carmel, Quinodoz Mathieu

机构信息

Ophthalmic Genetics Group, Institute of Molecular and Clinical Ophthalmology Basel (IOB), Basel 4031, Switzerland; Department of Ophthalmology, University of Basel, Basel 4031, Switzerland; Department of Genetics, Genomics and Cancer Sciences, University of Leicester, Leicester LE1 7RH, UK.

Ophthalmic Genetics Group, Institute of Molecular and Clinical Ophthalmology Basel (IOB), Basel 4031, Switzerland; Department of Ophthalmology, University of Basel, Basel 4031, Switzerland.

出版信息

Am J Hum Genet. 2025 Oct 2;112(10):2253-2265. doi: 10.1016/j.ajhg.2025.08.017. Epub 2025 Sep 16.

Abstract

Inherited retinal diseases (IRDs) are rare disorders, typically presenting as Mendelian traits, that result in stationary or progressive visual impairment. They are characterized by extensive genetic heterogeneity, possibly the highest among all human genetic diseases, as well as diverse inheritance patterns. Despite advances in gene discovery, limited understanding of gene function and challenges in accurately interpreting variants continue to hinder both molecular diagnosis and genetic research in IRDs. One key problem is the absence of a comprehensive and widely accepted catalog of disease-associated genes, which would ensure consistent genetic testing and reliable molecular diagnoses. With the rapid pace of IRD gene discovery, gene catalogs require frequent validation and updates to remain clinically and scientifically useful. To address these gaps, we developed RetiGene, an expert-curated gene atlas that integrates variant data, bulk and single-cell RNA sequencing, and functional annotations. Through the integration of diverse data sources, RetiGene supports candidate gene prioritization, functional studies, and therapeutic development in IRDs.

摘要

遗传性视网膜疾病(IRDs)是罕见疾病,通常表现为孟德尔性状,会导致静止性或进行性视力损害。它们的特点是具有广泛的遗传异质性,这可能是所有人类遗传疾病中最高的,并且遗传模式多样。尽管在基因发现方面取得了进展,但对基因功能的有限了解以及准确解释变异的挑战,仍然阻碍着IRDs的分子诊断和遗传研究。一个关键问题是缺乏全面且被广泛接受的疾病相关基因目录,而这将确保一致的基因检测和可靠的分子诊断。随着IRD基因发现的快速发展,基因目录需要频繁验证和更新,以保持临床和科学用途。为了填补这些空白,我们开发了RetiGene,这是一个由专家策划的基因图谱,整合了变异数据、批量和单细胞RNA测序以及功能注释。通过整合多种数据源,RetiGene支持IRDs中的候选基因优先级排序、功能研究和治疗开发。

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