Collagenopathy

胶原病
  • 文章类型: Case Reports
    Knobloch综合征是一种罕见的胶原病,其特征是严重的早发性近视,视网膜脱离,和枕骨脑膨出,由于COL18A1基因的双等位基因变化而出现各种其他表现。在这里,我们报道了一个中国家庭,有两个受影响的兄弟姐妹在产前出现枕骨脑膨出,婴儿发作性视网膜脱离,在儿童早期明显高度近视。在该家族中进行了四方全外显子组测序,并确定两个兄弟姐妹在COL18A1基因(NM_001379500.1)中都携带了新的复合杂合变体:在内含子8的共有受体剪接位点处的母系遗传变体c.1222-1G>A,以及父系遗传移码变体c.3931_3932delinsTp。两名患者在出生后不久就对枕骨脑膨出进行了成功的手术治疗。他们在姐姐的7岁和弟弟的4岁时具有正常的神经认知结果和良好的一般状况。弟弟在7个月大时出现了婴儿性视网膜脱离,而妹妹在7岁之前患有高度近视,没有视网膜脱离的迹象。该报告通过产前和产后发现扩展了Knobloch综合征的表型和基因型谱。
    Knobloch syndrome is a rare collagenopathy characterized by severe early onset myopia, retinal detachment, and occipital encephalocele with various additional manifestations due to biallelic changes in the COL18A1 gene. Here we reported a Chinese family with two affected siblings presented with antenatal occipital encephalocele, infantile onset retinal detachment, and pronounced high myopia at early childhood. Quartet whole exome sequencing was performed in this family and identified that both siblings carried novel compound heterozygous variants in the COL18A1 gene (NM_001379500.1): the maternally inherited variant c.1222-1G>A at the consensus acceptor splice site of intron 8, and the paternally inherited frameshift variant c.3931_3932delinsT p.(Gly1311Serfs*25) in the last exon. Both patients had successful surgical treatment for the occipital encephalocele soon after birth. They had normal neurocognitive outcome and good general conditions examined at the age of 7 years old for the elder sister and 4 years old for the younger brother. The younger brother developed infantile onset retinal detachment at 7 months of age while the sister had high myopia without signs of retinal detachment until 7 years old. This report expands the phenotype and genotype spectrum of Knobloch syndrome with antenatal and postnatal findings.
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