Department of Molecular Genetics & Dental Research Institute, School of Dentistry, Seoul National University, Seoul, Republic of Korea.
Department of Pediatric Dentistry & Dental Research Institute, School of Dentistry, Seoul National University, Seoul, Republic of Korea.
J Dent Res. 2020 Apr;99(4):410-418. doi: 10.1177/0022034520901708. Epub 2020 Jan 30.
Amelogenesis imperfecta (AI) is a collection of genetic disorders affecting the quality and/or quantity of tooth enamel. More than 20 genes are, so far, known to be responsible for this condition. In this study, we recruited 3 Turkish families with hypomaturation AI. Whole-exome sequence analyses identified disease-causing mutations in each proband, and these mutations cosegregated with the AI phenotype in all recruited members of each family. The AI-causing mutations in family 1 were a novel mutation [NM_182680.1:c.143T>C, p.(Leu48Ser)] in the proband and a novel homozygous mutation [NM_004771.3:c.616G>A, p.(Asp206Asn)] in the mother of the proband. Previously reported compound heterozygous mutations [NM_004771.3:c.103A>C, p.(Arg35=) and c.389C>T, p.(Thr130Ile)] caused the AI in family 2 and family 3. Minigene splicing analyses revealed that the missense mutation increased exonic definition of exon 4 and the synonymous mutation decreased exonic definition of exon 1. These mutations would trigger an alteration of exon usage during RNA splicing, causing the enamel malformations. These results broaden our understanding of molecular genetic pathology of tooth enamel formation.
遗传性牙釉质发育不全(AI)是一组影响牙釉质质量和/或数量的遗传疾病。到目前为止,已知有 20 多个基因与此病症有关。在这项研究中,我们招募了 3 个具有低成熟 AI 的土耳其家庭。全外显子组序列分析在每个先证者中确定了致病突变,这些突变在每个家庭中所有被招募的成员中与 AI 表型共分离。第 1 个家庭的 AI 致病突变是先证者中的一个新的错义突变 [NM_182680.1:c.143T>C, p.(Leu48Ser)] 和先证者母亲中的一个新的纯合突变 [NM_004771.3:c.616G>A, p.(Asp206Asn)]。先前报道的复合杂合错义突变 [NM_004771.3:c.103A>C, p.(Arg35=) 和 c.389C>T, p.(Thr130Ile)] 导致了第 2 个家庭和第 3 个家庭的 AI。小基因剪接分析表明,错义突变增加了外显子 4 的外显子定义,同义突变降低了外显子 1 的外显子定义。这些突变会在 RNA 剪接过程中引发外显子使用的改变,导致牙釉质畸形。这些结果拓宽了我们对牙齿釉质形成分子遗传病理学的理解。
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