Bakker E, Veenema H, Den Dunnen J T, van Broeckhoven C, Grootscholten P M, Bonten E J, van Ommen G J, Pearson P L
Department of Human Genetics, Sylvius Laboratories, Leiden, The Netherlands.
J Med Genet. 1989 Sep;26(9):553-9. doi: 10.1136/jmg.26.9.553.
In 288 Dutch and Belgian Duchenne and Becker muscular dystrophy families, the parental origin of 41 new deletion or duplication mutations was determined. Twenty seven of the new mutations occurred in the maternal X chromosome and nine in the grandmaternal and five in the grandpaternal X chromosome. The grandparental data are compatible with equal mutation rates for DMD in male and female X chromosomes. New mutations were defined by their presence in one or more progeny and absence in the lymphocytes of the mother or the grandparents. In one family a fraction of the maternal lymphocytes was found to carry the mutation, suggesting somatic mosaicism. In six cases out of 41, the mutation was transmitted more than once by a parent in whom the mutation was absent in lymphocytes, suggesting gonadal mosaicism as the explanation for the multiple transmission. Using our data for the recurrence of the mutations among the total of at risk haplotypes transmitted, we arrive at a recurrence risk of 14% for the at risk haplotype. The observation of this high risk of germinal mosaicism is crucially important for all physicians counselling females in DMD families. Recently, germinal mosaicism has been observed also in a number of other X linked and autosomal disorders. The implications and appropriate diagnostic precautions are discussed.
在288个荷兰和比利时的杜兴氏和贝克氏肌营养不良症家庭中,确定了41个新的缺失或重复突变的亲本来源。其中27个新突变发生在母本X染色体上,9个发生在祖母的X染色体上,5个发生在祖父的X染色体上。祖父母的数据与男性和女性X染色体上DMD的突变率相等相符。新突变的定义是在一个或多个后代中出现,而在母亲或祖父母的淋巴细胞中不存在。在一个家庭中,发现一部分母本淋巴细胞携带该突变,提示体细胞嵌合现象。在41个案例中有6个案例中,该突变由淋巴细胞中不存在该突变的亲本多次传递,提示生殖腺嵌合现象是多次传递的原因。利用我们的数据计算在总共传递的风险单倍型中突变的复发率,得出风险单倍型的复发风险为14%。对于所有为杜兴氏肌营养不良症家庭中的女性提供咨询的医生来说,观察到这种高生殖腺嵌合风险至关重要。最近,在许多其他X连锁和常染色体疾病中也观察到了生殖腺嵌合现象。文中讨论了其影响和适当的诊断预防措施。