Burusnukul Prinyarat, de los Reyes Emily C
Department of Pediatrics and Neurology, Nationwide Children's Hospital, The Ohio State University, Columbus, Ohio, USA.
J Child Neurol. 2009 Apr;24(4):482-6. doi: 10.1177/0883073808324539. Epub 2009 Feb 2.
Autosomal inherited mitochondrial diseases have been of increasing interest among clinicians and mitochondrial research groups because these diseases are caused through a secondary effect on the mitochondrial DNA. It was thought that the genetic stability of mitochondrial DNA relies on the accuracy of DNA polymerase gamma. Mutations of DNA polymerase gamma 1 gene (MIM# 174763) have been shown to be a cause of mitochondrial disorders associated with Mendelian disorders characterized by multiple mitochondrial DNA deletions or depletions. To date, several clinical phenotypes associated with polymerase gamma mutation have been reported presenting in both adults and children. We present 3 children in whom were found to have reported pathogenic DNA polymerase gamma 1 mutations: heterozygous p.G517V in 2 half siblings and heterozygous p.T251I and p.P587L in the other. The aim of this communication is to report 3 pediatric cases associated with DNA polymerase gamma 1 mutations to augment the expanding clinical phenotype that has been previously reported.
常染色体遗传的线粒体疾病越来越受到临床医生和线粒体研究团队的关注,因为这些疾病是由对线粒体DNA的继发性影响引起的。人们认为线粒体DNA的遗传稳定性依赖于DNA聚合酶γ的准确性。DNA聚合酶γ1基因(MIM# 174763)的突变已被证明是与孟德尔疾病相关的线粒体疾病的一个病因,其特征为多个线粒体DNA缺失或耗竭。迄今为止,已报道了几种与聚合酶γ突变相关的临床表型,在成人和儿童中均有出现。我们报告了3名儿童,他们被发现携带已报道的致病性DNA聚合酶γ1突变:2名同父异母的兄弟姐妹为杂合子p.G517V,另一名为杂合子p.T251I和p.P587L。本通讯的目的是报告3例与DNA聚合酶γ1突变相关的儿科病例,以补充先前报道的不断扩大的临床表型。