Yeowell H N, Walker L C
Duke University Medical Center, Durham, NC 27710, USA.
Proc Assoc Am Physicians. 1999 Jan-Feb;111(1):57-62. doi: 10.1046/j.1525-1381.1999.09112.x.
We have performed the first prenatal assessment of clinical phenotype in a family affected by Ehlers-Danlos syndrome type VI (EDS VI), an inherited collagen disorder, by screening the fetal DNA for mutations in the lysyl hydroxylase (LH) gene. We have previously reported that the affected child in this family is compound heterozygous for mutations in the LH gene. One allele has a paternally inherited C1557 to G change that codes for a premature stop codon (Y511X) in exon 14 and the other allele has a deletion of exon 5 that results from a maternally inherited mutation in the consensus donor splice site of intron 5. To perform the prenatal diagnosis, we sequenced genomic DNA isolated from cultured chorionic villus cells at 10 weeks of gestation. One allele had the maternally inherited gt --> at splice-site mutation in exon 5, and the other paternally inherited allele was normal. As EDS VI is a recessive disorder, we predicted that although a carrier, the baby should be unaffected. This conclusion, which was supported by a normal level of LH activity in the chorionic villus cells, was confirmed by the birth of a healthy unaffected baby.
我们通过筛查胎儿DNA中赖氨酰羟化酶(LH)基因的突变,对一个受VI型埃勒斯-当洛综合征(EDS VI,一种遗传性胶原蛋白疾病)影响的家庭进行了首次临床表型的产前评估。我们之前报道过,这个家庭中的患病儿童是LH基因的复合杂合突变。一个等位基因有一个从父亲遗传来的C1557到G的变化,该变化在外显子14中编码一个提前终止密码子(Y511X),另一个等位基因有外显子5的缺失,这是由母亲遗传的内含子5共有供体剪接位点突变导致的。为了进行产前诊断,我们对妊娠10周时从培养的绒毛膜绒毛细胞中分离的基因组DNA进行了测序。一个等位基因有母亲遗传的外显子5剪接位点突变gt --> at,另一个从父亲遗传的等位基因是正常的。由于EDS VI是一种隐性疾病,我们预测,尽管是携带者,但婴儿应该不会患病。绒毛膜绒毛细胞中LH活性水平正常支持了这一结论,一个健康未患病婴儿的出生证实了这一点。