MedElevator Войти в исследования
← Поиск публикаций

DOI 10.1016/j.xhgg.2024.100379

Медицинская библиотека · HGG advances · 2024

Expanding the phenotypic spectrum of CSNK2A1-associated Okur-Chung neurodevelopmental syndrome.

Ramadesikan Swetha, Showpnil Iftekhar A, Marhabaie Mohammad, Daley Allison, Varga Elizabeth A, Gurusamy Umamaheswaran, Pastore Matthew T, Sites Emily R, Manickam Murugu, Bartholomew Dennis W, Hunter Jesse M, White Peter, Wilson Richard K, Stottmann Rolf W, Koboldt Daniel C

СтатьяКлинический случайПеревод в очереди

Аннотация

De novo variants in CSNK2A1 cause autosomal dominant Okur-Chung neurodevelopmental syndrome (OCNDS). OCNDS has an evolving clinical phenotype predominantly characterized by intellectual disability, global delays, dysmorphic features, and immunological manifestations. Microcephaly, defined as a small head circumference, is not widely recognized as a classical clinical presentation. Here, we describe four individuals from three unrelated families who shared several clinical features characteristic of an underlying syndromic neurodevelopmental condition. Trio clinical exome and research genome sequencing revealed that all affected individuals had heterozygous pathogenic missense variants in CSNK2A1. Two variants (c.468T>A p.Asp156Glu and c.149A>G p.Tyr50Cys) were de novo and previously reported, but the third variant (c.137G>T p.Gly46Val) is novel and segregated in two affected individuals in a family. This adds to growing evidence of inherited disease-causing variants in CSNK2A1, an observation reported only twice previously. A detailed phenotypic analysis of our cohort together with those individuals reported in the literature revealed that OCNDS individuals, on average, have a smaller head circumference with one-third presenting with microcephaly. We also show that the incidence of microcephaly is significantly correlated with the location of the variant in the encoded protein. Our findings suggest that small head circumference is a common but under-recognized feature of OCNDS, which may not be apparent at birth.

Для цитирования: Ramadesikan Swetha, Showpnil Iftekhar A, Marhabaie Mohammad, Daley Allison, Varga Elizabeth A, Gurusamy Umamaheswaran, Pastore Matthew T, Sites Emily R, Manickam Murugu, Bartholomew Dennis W, Hunter Jesse M, White Peter, Wilson Richard K, Stottmann Rolf W, Koboldt Daniel C. Expanding the phenotypic spectrum of CSNK2A1-associated Okur-Chung neurodevelopmental syndrome.. HGG advances. 2024. DOI: 10.1016/j.xhgg.2024.100379.

Предметные рубрики MeSH

Подготовка и перевод публикации
Обработка статьи · этапы, ошибки и управление
Загрузка состояния…

История · было → стало

Состояние обновляется каждые 5 секунд, пока блок открыт. Процент показывается только там, где известен объём работы. Пауза останавливает продолжение этапа; текущий запрос к источнику может завершиться.

Тип публикации указан по данным источника. Уровень доказательности автоматически не присваивается.