Kieffer Emmanuelle, Nicod Jean-Christophe, Gardes Nathalie, Kastner Claire, Becker Nicolas, Celebi Catherine, Pirrello Olivier, Rongières Catherine, Koscinski Isabelle, Gosset Philippe, Moutou Céline
Laboratoire de Diagnostic Préimplantatoire, Hôpitaux Universitaires de Strasbourg, site du CMCO, Strasbourg, France.
Laboratoire de Biologie de la Reproduction, Hôpitaux Universitaires de Strasbourg, site du CMCO, Strasbourg, France.
Eur J Hum Genet. 2016 Feb;24(2):221-7. doi: 10.1038/ejhg.2015.96. Epub 2015 May 13.
Fragile X syndrome (FraX) is caused by the expansion of an unstable CGG repeat located in the Fragile X mental retardation 1 gene (FMR1) gene. Preimplantation genetic diagnosis (PGD) can be proposed to couples at risk of transmitting the disease, that is, when the female carries a premutation or a full mutation. We describe two new single-cell, single-round multiplex PCR for indirect and direct diagnosis of FraX on biopsied embryos. These tests include five unpublished, highly heterozygous simple sequence repeats, and the co-amplification of non-expanded CGG repeats for the direct test. Heterozygosity of the new markers ranged from 69 to 81%. The mean rate of non-informative marker included in the tests was low (26% and 23% for the new indirect and direct tests, respectively). This strategy allows offering a PGD for FraX to 96% of couples requesting it in our centre. A conclusive genotype was obtained in all cells with a rate of cells presenting an allele dropout ranging from 17% for the indirect test to 26% for the direct test. The new indirect test was applied for eight PGD cycles: 32 embryos were analysed, 9 were transferred and 3 healthy babies were born. By multiplexing these highly informative markers, robustness of the diagnosis is improved and the loss of potentially healthy embryos (because they are non-diagnosed or misdiagnosed) is limited. This may increase the chances of success of couples requesting a PGD for FraX, in particular, when premature ovarian insufficiency in premutated women leads to a reduced number of embryos available for analysis.
脆性X综合征(FraX)由位于脆性X智力低下1基因(FMR1)中的不稳定CGG重复序列扩增引起。对于有疾病遗传风险的夫妇,即女性携带前突变或全突变时,可建议进行植入前基因诊断(PGD)。我们描述了两种新的单细胞、单轮多重PCR方法,用于对活检胚胎进行脆性X综合征的间接和直接诊断。这些检测包括五个未发表的、高度杂合的简单序列重复序列,以及用于直接检测的非扩增CGG重复序列的共扩增。新标记的杂合度范围为69%至81%。检测中包含的无信息标记的平均比例较低(新的间接检测和直接检测分别为26%和23%)。这种策略使我们中心96%要求进行脆性X综合征PGD的夫妇能够进行该检测。在所有细胞中均获得了决定性的基因型,等位基因脱失的细胞比例为间接检测17%,直接检测26%。新的间接检测应用于8个PGD周期:分析了32个胚胎,移植了9个,出生了3个健康婴儿。通过复用这些信息丰富的标记,提高了诊断的稳健性,减少了潜在健康胚胎(因未诊断或误诊)的损失。这可能会增加要求进行脆性X综合征PGD的夫妇成功的机会,特别是当携带前突变的女性出现卵巢早衰导致可供分析的胚胎数量减少时。