Karstensen Helena Gásdal, Rendtorff Nanna Dahl, Hindbæk Lone Sandbjerg, Colombo Roberto, Stein Amelie, Birkebæk Niels Holtum, Hartmann-Petersen Rasmus, Lindorff-Larsen Kresten, Højland Allan Thomas, Petersen Michael Bjørn, Tranebjærg Lisbeth
The Kennedy Center, Department of Clinical Genetics, Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark.
The Kennedy Center, Department of Clinical Genetics, Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark.
Eur J Med Genet. 2020 Mar;63(3):103733. doi: 10.1016/j.ejmg.2019.103733. Epub 2019 Aug 23.
Biallelic variants in HARS2 have been associated with Perrault syndrome, characterized by sensorineural hearing impairment and premature ovarian insufficiency. Here we report three novel families, compound heterozygous for missense variants in HARS2 identified by next-generation sequencing, namely c.172A > G (p.Lys58Glu) and c.448C > T (p.Arg150Cys) identified in two sisters aged 13 and 16 years and their older brother, c.448C > T (p.Arg150Cys) and c.980G > A (p.Arg327Gln) identified in a seven year old girl, and finally c.137T > A (p.Leu46Gln) and c.259C > T (p.Arg87Cys) identified in a 32 year old woman. Clinically, all five individuals presented with early onset, rapidly progressive hearing impairment. Whereas the oldest female fulfilled the criteria of Perrault syndrome, the three younger females, aged 7, 13 and 16, all had apparently normal ovarian function, apart from irregular menstrual periods in the oldest female at age 16. The present report expands the list of HARS2 variants and helps gain further knowledge to the phenotype.
HARS2基因的双等位基因变异与佩罗特综合征相关,其特征为感音神经性听力障碍和卵巢早衰。在此,我们报告三个新的家系,通过下一代测序鉴定出HARS2基因错义变异的复合杂合子,即13岁和16岁的两姐妹及其哥哥中鉴定出的c.172A>G(p.Lys58Glu)和c.448C>T(p.Arg150Cys),一名7岁女孩中鉴定出的c.448C>T(p.Arg150Cys)和c.980G>A(p.Arg327Gln),以及一名32岁女性中鉴定出的c.137T>A(p.Leu46Gln)和c.259C>T(p.Arg87Cys)。临床上,所有五名个体均表现为早发性、快速进展性听力障碍。虽然最年长的女性符合佩罗特综合征的标准,但三名较年轻的女性,年龄分别为7岁、13岁和16岁,除了16岁最年长女性月经不规律外,卵巢功能显然正常。本报告扩展了HARS2变异的列表,并有助于进一步了解该表型。