Yamagishi Hiroyuki
Department of Pediatrics and Molecular Biology, University of Texas Southwestern Medical Center, Dallas 75390-9148, USA.
Keio J Med. 2002 Jun;51(2):77-88. doi: 10.2302/kjm.51.77.
The 22q11.2 deletion syndrome (22q11DS) encompasses DiGeorge syndrome, velo-cardio-facial syndrome and conotruncal anomaly face syndrome and is due to a microdeletion of chromosome 22q11.2. This is the most frequent known interstitial deletion found in human with an incidence of 1 in 4,000 live births. A large number of clinical findings have been reported in affected patients, including cardiac defects, characteristic facial features, thymic hypoplasia, cleft palate, hypoparathyroidism, learning difficulties and psychiatric disorders. A comprehensive evaluation and follow-up program is necessary for patients with 22q11DS. A striking aspect of the 22q11DS phenotype is its variability, the basis of which remains unclear, and no phenotype-genotype correlation has been made. The structures primarily affected in patients with 22q11DS are derivatives of the embryonic pharyngeal arches and pouches suggesting that haploinsufficiency of the gene(s) on the deleted region, spanning 2-3 Mb, is important in pharyngeal arch/pouch development. Extensive gene searches have been successful in identifying more than 30 genes in the deleted segment. Although standard positional cloning has failed to demonstrate a role for any of these genes in the syndrome, the use of experimental animal models and advanced genome manipulation technologies in mice have been providing an insight into the developmental role of some of these genes, including TBXI. In this review, the clinical features and management of patients with 22q11DS are integrated with our current understanding of the embryological and molecular basis of this syndrome, as presented at the 1235th Meeting of The Keio Medical Society.
22q11.2缺失综合征(22q11DS)包括迪格奥尔格综合征、腭心面综合征和圆锥动脉干异常面容综合征,是由22号染色体q11.2区域的微缺失引起的。这是人类已知最常见的间质性缺失,活产发病率为1/4000。大量临床发现已在受影响患者中报道,包括心脏缺陷、特征性面部特征、胸腺发育不全、腭裂、甲状旁腺功能减退、学习困难和精神障碍。对于22q11DS患者,全面的评估和随访计划是必要的。22q11DS表型的一个显著特点是其变异性,其基础尚不清楚,且未建立表型-基因型相关性。22q11DS患者主要受影响的结构是胚胎咽弓和咽囊的衍生物,这表明缺失区域(跨度为2-3 Mb)上基因的单倍剂量不足在咽弓/咽囊发育中很重要。广泛的基因搜索已成功在缺失片段中鉴定出30多个基因。尽管标准的定位克隆未能证明这些基因中的任何一个在该综合征中起作用,但在小鼠中使用实验动物模型和先进的基因组操作技术为深入了解其中一些基因(包括TBXI)的发育作用提供了线索。在本次综述中,22q11DS患者的临床特征和管理与我们目前对该综合征胚胎学和分子基础的理解相结合,这些内容在庆应义塾医学会第1235次会议上有所介绍。
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