A Heterozygous NDUFV1 Variant Aggravates Mitochondrial Complex I Deficiency in a Family with a Homoplasmic ND1 Variant.

Baertling, Fabian; Sánchez-Caballero, Laura; van den Brand, Mariël A M; Distelmaier, Felix; Janssen, Mirian C H; Rodenburg, Richard J T; Smeitink, Jan A M; Nijtmans, Leo G J · J Pediatr · 2018

case_report · Level V

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Abstract

We demonstrate that a heterozygous nuclear variant in the gene encoding mitochondrial complex I subunit NDUFV1 aggravates the cellular phenotype in the presence of a mitochondrial DNA variant in complex I subunit ND1. Our findings suggest that heterozygous variants could be more significant in inherited mitochondrial diseases than hitherto assumed.

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