Gicquel Christine, Rossignol Sylvie, Cabrol Sylvie, Houang Muriel, Steunou Virginie, Barbu Véronique, Danton Fabienne, Thibaud Nathalie, Le Merrer Martine, Burglen Lydie, Bertrand Anne-Marie, Netchine Irène, Le Bouc Yves
Laboratoire d'Explorations Fonctionnelles Endocriniennes, Inserm U515 et UPMC Paris 6, Hôpital Armand Trousseau, AP-HP, 26 avenue Arnold Netter, 75012 Paris, France.
Nat Genet. 2005 Sep;37(9):1003-7. doi: 10.1038/ng1629. Epub 2005 Aug 7.
Silver-Russell syndrome (SRS, OMIM 180860) is a congenital disorder characterized by severe intrauterine and postnatal growth retardation, dysmorphic facial features and body asymmetry. SRS is genetically heterogenous with maternal uniparental disomy with respect to chromosome 7 occurring in approximately 10% of affected individuals. Given the crucial role of the 11p15 imprinted region in the control of fetal growth, we hypothesized that dysregulation of genes at 11p15 might be involved in syndromic intrauterine growth retardation. We identified an epimutation (demethylation) in the telomeric imprinting center region ICR1 of the 11p15 region in several individuals with clinically typical SRS. This epigenetic defect is associated with, and probably responsible for, relaxation of imprinting and biallelic expression of H19 and downregulation of IGF2. These findings provide new insight into the pathogenesis of SRS and strongly suggest that the 11p15 imprinted region, in addition to those of 7p11.2-p13 and 7q31-qter, is involved in SRS.
Silver-Russell综合征(SRS,OMIM 180860)是一种先天性疾病,其特征为严重的宫内和出生后生长迟缓、面部畸形特征及身体不对称。SRS在遗传上具有异质性,约10%的受影响个体存在母源单亲二体7号染色体。鉴于11p15印记区域在胎儿生长控制中的关键作用,我们推测11p15基因的失调可能与综合征性宫内生长迟缓有关。我们在几名临床典型SRS个体的11p15区域端粒印记中心区域ICR1中发现了一种表观突变(去甲基化)。这种表观遗传缺陷与印记放松、H19双等位基因表达以及IGF2下调相关,且可能是其原因。这些发现为SRS的发病机制提供了新见解,并强烈表明除了7p11.2-p13和7q31-qter区域外,11p15印记区域也与SRS有关。