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Eur J Med Genet ; 63(4): 103821, 2020 Apr.
Artigo em Inglês | MEDLINE | ID: mdl-31778857

RESUMO

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.


Assuntos
DNA Mitocondrial/genética , DNA Polimerase Dirigida por DNA/genética , Transtornos dos Movimentos/genética , Atrofia Óptica/genética , Polineuropatias/genética , Insuficiência Ovariana Primária/genética , Adulto , Feminino , Humanos , Mutação de Sentido Incorreto
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