Tamhankar P M, Iyer S, Sanghavi S, Khopkar U
Genetic Research Center, National Institute for Research in Reproductive Health (NIRRH), Parel, Mumbai, Maharashtra, India.
J Postgrad Med. 2014 Jul-Sep;60(3):332-4. doi: 10.4103/0022-3859.138826.
Chanarin-Dorfman syndrome (CDS) is a multisystem, autosomal recessive genetic disorder characterized by congenital non-bullous ichthyosiform erythroderma with accumulation of lipid droplets in granulocytes and basal keratinocytes. An 18-month-old female child presented with typical dermatological features of CDS. She was born as a collodion baby. Liver biopsy showed micronodular cirrhosis along with macrovesicular hepatic steatosis. Sequencing of all exons and exon-intron boundaries of the ABHD5 gene showed that the patient was homozygous for a novel mutation g.24947delG (c.773 + 1delG) in intron 5. This is the first Indian child with mutation proven CDS.
钱纳林-多夫曼综合征(CDS)是一种多系统常染色体隐性遗传病,其特征为先天性非大疱性鱼鳞病样红皮病,伴有粒细胞和基底角质形成细胞中脂滴积聚。一名18个月大的女童表现出CDS典型的皮肤特征。她出生时为胶样婴儿。肝脏活检显示微结节性肝硬化以及大泡性肝脂肪变性。对ABHD5基因的所有外显子和外显子-内含子边界进行测序,结果显示该患者在第5内含子中存在一种新的突变g.24947delG(c.773 + 1delG)的纯合子。这是首例经突变证实的印度CDS患儿。