| Literature DB >> 31778857 |
Petra Dosekova1, Andrzej Dubiel2, Anna Karlowicz2, Szymon Zietkiewicz2, Malgorzata Rydzanicz3, Viera Habalova4, Victor Murcia Pienkowski5, Miriam Skirkova6, Vladimir Han7, Alexandra Mosejova7, Zuzana Gdovinova7, Magdalena Kaliszewska8, Katarzyna Tońska8, Michal R Szymanski2, Matej Skorvanek7, Rafal Ploski9.
Abstract
POLG2 associated disorders belong to the group of mitochondrial DNA (mtDNA) diseases and present with a heterogeneous clinical spectrum, various age of onset, and disease severity. We report a 39-year old female presenting with childhood-onset and progressive neuroophthalmic manifestation with optic atrophy, mixed polyneuropathy, spinal and cerebellar ataxia and generalized chorea associated with mtDNA depletion. Whole-exome sequencing identified an ultra-rare homozygous missense mutation located at Chr17: 062474101-C > A (p.Asp433Tyr) in nuclear POLG2 gene encoding PolγB, an accessory subunits of mitochondrial polymerase γ responsible for mtDNA replication. The healthy parents and 2 sisters of the patient were heterozygous for the variant. To our best knowledge, this is the first case of homozygous variant in the POLG2 gene resulting in mitochondrial depletion syndrome in an adult patient and its clinical manifestations extend the clinical spectrum of POLG2 associated diseases.Entities:
Keywords: Ataxia; Chorea; Homozygous; Mitochondrial DNA depletion; Optic atrophy; POLG2; Polyneuropathy; Polγ; Premature ovarian failure
Year: 2019 PMID: 31778857 DOI: 10.1016/j.ejmg.2019.103821
Source DB: PubMed Journal: Eur J Med Genet ISSN: 1769-7212 Impact factor: 2.708