Sir Run Run Hospital, Nanjing Medical University, Nanjing, China.
Department of Ophthalmology, Children's Hospital of Nanjing Medical University, Nanjing, China.
J Hum Genet. 2021 May;66(5):543-548. doi: 10.1038/s10038-020-00872-z. Epub 2020 Nov 11.
Foveal hypoplasia is the major cause of visual loss. Here we report an isolated foveal hypoplasia patient without nystagmus. It is very rare, and its etiology is not completely understood. Using whole-exome sequencing and foveal hypoplasia-related gene filtering from a family with two generations, we identified a novel variant c.859T>C (p.S287P) and a rare non-frameshift variant c.229_230insGGG (p.Arg77_Glu78insGly) in the tyrosinase (TYR) gene that co-segregated in the affected member of this family. The compound heterozygous variants inherited in the proband were confirmed by Sanger sequencing and predicted from in silico studies to have an effect on protein function. In conclusion, our finding extends the spectrum of TYR variants and supports the important role of TYR in the development of eyes.
黄斑发育不良是导致视力丧失的主要原因。在这里,我们报告了一例孤立性黄斑发育不良患者,无眼球震颤。这种情况非常罕见,其病因尚不完全清楚。通过对一个有两代人家庭的全外显子组测序和与黄斑发育不良相关基因的筛选,我们在酪氨酸酶(TYR)基因中发现了一个新的 c.859T>C(p.S287P)变异和一个罕见的非移码变异 c.229_230insGGG(p.Arg77_Glu78insGly),该变异在该家庭的受影响成员中共同遗传。通过 Sanger 测序和计算机研究预测,杂合子变体在该先证者中被证实对蛋白功能有影响。总之,我们的发现扩展了 TYR 变异谱,并支持 TYR 在眼睛发育中的重要作用。