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在两个患有常染色体隐性端粒生物学障碍的家族中鉴定双等位基因POLA2变异体。

Identification of biallelic POLA2 variants in two families with an autosomal recessive telomere biology disorder.

作者信息

Kvarnung Malin, Pettersson Maria, Chun-On Pattra, Rafati Maryam, McReynolds Lisa J, Norberg Anna, Moura Pedro Luis, Pesonen Ida, Chaireti Roza, Grönros Söderholm Boa, Burlin Julia, Rydén Jenny, Lindberg Eva Hellström, Giri Neelam, Savage Sharon A, Agarwal Suneet, Nordgren Ann, Tesi Bianca

机构信息

Department of Molecular Medicine and Surgery, Karolinska Institutet, Stockholm, Sweden.

Clinical Genetics and Genomics, Karolinska University Hospital, Stockholm, Sweden.

出版信息

Eur J Hum Genet. 2025 May;33(5):580-587. doi: 10.1038/s41431-024-01722-8. Epub 2024 Nov 30.

Abstract

POLA2 encodes the accessory subunit of DNA polymerase α (polα)/primase, which is crucial for telomere C-strand fill-in. Incomplete fill-in of the C-rich telomeric strand after DNA replication has been proposed as a mechanism for Coats plus syndrome, a phenotype within the broader spectrum of telomere biology disorders (TBD). Coats plus syndrome has so far been associated with pathogenic variants in POT1, CTC1, and STN1. Here we report the findings of biallelic deleterious rare variants in POLA2 gene detected by whole genome sequencing and segregation analysis in five young adults from two unrelated families. All five individuals displayed abnormally short telomeres and a clinical phenotype suggesting a TBD disorder with Coats plus features including retinal and gastrointestinal telangiectasias. Our results suggest POLA2 as a novel autosomal recessive gene for a TBD with Coats plus features.

摘要

POLA2编码DNA聚合酶α(polα)/引发酶的辅助亚基,这对于端粒C链填充至关重要。DNA复制后富含C的端粒链填充不完全被认为是科茨加综合征的一种机制,科茨加综合征是端粒生物学障碍(TBD)更广泛谱系中的一种表型。迄今为止,科茨加综合征已与POT1、CTC1和STN1中的致病变异相关。在此,我们报告了通过全基因组测序和分离分析在来自两个无关家族的五名年轻成年人中检测到的POLA2基因双等位基因有害罕见变异的研究结果。所有五名个体均表现出异常短的端粒以及提示TBD疾病且具有包括视网膜和胃肠道毛细血管扩张等科茨加特征的临床表型。我们的结果表明,POLA2是一种具有科茨加特征的TBD的新型常染色体隐性基因。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/8122/12048608/08906ee289b3/41431_2024_1722_Fig1_HTML.jpg

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