Steffann J, Frydman N, Gigarel N, Burlet P, Ray P F, Fanchin R, Feyereisen E, Kerbrat V, Tachdjian G, Bonnefont J-P, Frydman R, Munnich A
J Med Genet. 2006 Mar;43(3):244-7. doi: 10.1136/jmg.2005.032326. Epub 2005 Sep 9.
Diseases arising from mitochondrial DNA (mtDNA) mutations are usually serious pleiotropic disorders with maternal inheritance. Owing to the high recurrence risk in the progeny of carrier females, "at-risk" couples often ask for prenatal diagnosis. However, reliability of such practices remains under debate. Preimplantation diagnosis (PGD), a theoretical alternative to conventional prenatal diagnosis, requires that the mutant load measured in a single cell from an eight cell embryo accurately reflects the overall heteroplasmy of the whole embryo, but this is not known to be the case.
To investigate the segregation of an mtDNA length polymorphism in blastomeres of 15 control embryos from four unrelated couples, the NARP mutation in blastomeres of three embryos from a carrier of this mutation.
Variability of the mtDNA polymorphism heteroplasmy among blastomeres from each embryo was limited, ranging from zero to 19%, with a mean of 7%. PGD for the neurogenic ataxia retinitis pigmentosa (NARP) mtDNA mutation (8993T-->G) was therefore carried out in the carrier mother of an affected child. One of three embryos was shown to carry 100% of mutant mtDNA species while the remaining two were mutation-free. These two embryos were transferred, resulting in a singleton pregnancy with delivery of a healthy child.
This PGD, the first reported for a mtDNA mutation, illustrates the skewed meiotic segregation of the NARP mtDNA mutation in early human development. However, discrepancies between the segregation patterns of the NARP mutation and the HV2 polymorphism indicate that a particular mtDNA nucleotide variant might differentially influenced the mtDNA segregation, precluding any assumption on feasibility of PGD for other mtDNA mutations.
由线粒体DNA(mtDNA)突变引起的疾病通常是具有母系遗传的严重多效性疾病。由于携带突变的女性后代复发风险高,“高危”夫妇常要求进行产前诊断。然而,此类做法的可靠性仍存在争议。植入前诊断(PGD)是传统产前诊断的一种理论替代方法,它要求从八细胞胚胎的单个细胞中测得的突变负荷准确反映整个胚胎的总体异质性,但目前尚不清楚是否如此。
研究来自四对无关夫妇的15个对照胚胎的卵裂球中线粒体DNA长度多态性的分离情况,以及来自该突变携带者的三个胚胎的卵裂球中的NARP突变情况。
每个胚胎的卵裂球中线粒体DNA多态性异质性的变异性有限,范围从零到19%,平均为7%。因此,对一名患病儿童的携带者母亲进行了神经源性共济失调视网膜色素变性(NARP)线粒体DNA突变(8993T→G)的植入前诊断。三个胚胎中的一个显示携带100%的突变型线粒体DNA,而其余两个没有突变。这两个胚胎被移植,结果单胎妊娠并分娩出一个健康的婴儿。
这例首次报道的针对线粒体DNA突变的植入前诊断,说明了NARP线粒体DNA突变在人类早期发育中的减数分裂分离偏斜。然而,NARP突变和HV2多态性的分离模式之间的差异表明,特定的线粒体DNA核苷酸变异可能对线粒体DNA分离有不同影响,排除了对其他线粒体DNA突变进行植入前诊断可行性的任何假设。