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DOI 10.1002/ajmg.a.38615

Медицинская библиотека · American journal of medical genetics. Part A · 2018

Further delineation of Temtamy syndrome of corpus callosum and ocular abnormalities.

Alrakaf Laila, Al-Owain Mohammed A, Busehail Maryam, Alotaibi Maha A, Monies Dorota, Aldhalaan Hesham M, Alhashem Amal, Al-Hassnan Zuhair N, Rahbeeni Zuhair A, Murshedi Fathiya Al, Ani Nadia Al, Al-Maawali Almundher, Ibrahim Niema A, Abdulwahab Firdous M, Alsagob Maysoon, Hashem Mais O, Ramadan Wafaa, Abouelhoda Mohamed, Meyer Brian F, Kaya Namik, Maddirevula Sateesh, Alkuraya Fowzan S

СтатьяПоддержка исследования: вне правительства СШАПеревод в очереди

Аннотация

Temtamy syndrome is a syndromic form of intellectual disability characterized by ocular involvement, epilepsy and dysgenesis of the corpus callosum. After we initially mapped the disease to C12orf57, we noted a high carrier frequency of an ancient startloss founder mutation [c.1A>G; p.M1?] in our population, and variable phenotypic expressivity in newly identified cases. This study aims to combine 33 previously published patients with 23 who are described here for the first time to further delineate the phenotype of this syndrome. In addition to the known p.M1? founder, we describe four novel homozygous variants, thus increasing the number of Temtamy syndrome-related C12orf57 variants to seven, all but one predicted to be loss of function. While all patients presented with intellectual disability/developmental delay, the frequency of other phenotypic features was variable: 73.2% (41/56) had epilepsy, 63% (34/54) had corpus callosal abnormalities, 14.5% (8/55) had coloboma, and 16.4% (9/55) had microphthalmia. Our analysis also revealed a high frequency of less recognized features such as congenital heart disease (51.4%), and brain white matter abnormalities (38%, 19/50). We conclude that C12orf57 variants should be considered in the etiology of developmental delay/intellectual disability, even when typical syndromic features are lacking, especially in those who trace their ancestry to Saudi Arabia where a founder C12orf57 mutation is among the most common recessive causes of intellectual disability.

Для цитирования: Alrakaf Laila, Al-Owain Mohammed A, Busehail Maryam, Alotaibi Maha A, Monies Dorota, Aldhalaan Hesham M, Alhashem Amal, Al-Hassnan Zuhair N, Rahbeeni Zuhair A, Murshedi Fathiya Al, Ani Nadia Al, Al-Maawali Almundher, Ibrahim Niema A, Abdulwahab Firdous M, Alsagob Maysoon, Hashem Mais O, Ramadan Wafaa, Abouelhoda Mohamed, Meyer Brian F, Kaya Namik, Maddirevula Sateesh, Alkuraya Fowzan S. Further delineation of Temtamy syndrome of corpus callosum and ocular abnormalities.. American journal of medical genetics. Part A. 2018. DOI: 10.1002/ajmg.a.38615.

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