Department of Neurology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania.
Department of Human Genetics and Hussman Institute for Human Genomics, University of Miami, Miami, Florida.
J Peripher Nerv Syst. 2019 Jun;24(2):213-218. doi: 10.1111/jns.12313. Epub 2019 Apr 10.
We report on two patients, with different POLG mutations, in whom axonal neuropathy dominated the clinical picture. One patient presented with late onset sensory axonal neuropathy caused by a homozygous c.2243G>C (p.Trp748Ser) mutation that resulted from uniparental disomy of the long arm of chromosome 15. The other patient had a complex phenotype that included early onset axonal Charcot-Marie-Tooth disease (CMT) caused by compound heterozygous c.926G>A (p.Arg309His) and c.2209G>C (p.Gly737Arg) mutations.
我们报告了两名具有不同 POLG 突变的患者,他们的临床表现以轴索性神经病为主。一名患者表现为晚发性感觉性轴索性神经病,由 15 号染色体长臂单亲二体导致的纯合 c.2243G>C(p.Trp748Ser)突变引起。另一名患者的表型复杂,包括早发性轴索性夏科-马里-图什病(CMT),由复合杂合 c.926G>A(p.Arg309His)和 c.2209G>C(p.Gly737Arg)突变引起。