Pathogenic mutations in the 5' untranslated region of BCS1L mRNA in mitochondrial complex III deficiency

Mitochondrion. 2009 Sep;9(5):299-305. doi: 10.1016/j.mito.2009.04.001. Epub 2009 Apr 21.

Abstract

Mutations in the assembly chaperone BCS1L constitute a major cause of mitochondrial complex III deficiency. We studied the presence of BCS1L mutations in a complex III-deficient patient with metabolic acidosis, liver failure, and tubulopathy. A previously reported mutation, p.R56X, was identified in one BCS1L allele, and two novel heterozygous mutations, g.1181A>G and g.1164C>G, were detected in the second allele. The g.1181A>G mutation generated an alternative splicing site in the BCS1L transcript, causing a 19-nucleotides deletion in its 5'UTR region. Decreased BCS1L mRNA and protein levels, and a respiratory chain complex III assembly impairment, determine a pathogenic role for the novel BCS1L mutations.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • 5' Untranslated Regions / genetics*
  • ATPases Associated with Diverse Cellular Activities
  • Base Sequence
  • Electron Transport Complex III / biosynthesis
  • Electron Transport Complex III / deficiency*
  • Electron Transport Complex III / genetics
  • Female
  • Humans
  • Infant, Newborn
  • Male
  • Models, Molecular
  • Molecular Sequence Data
  • Nucleic Acid Conformation
  • Point Mutation*
  • Pregnancy
  • RNA, Messenger / genetics*
  • Sequence Analysis, DNA

Substances

  • 5' Untranslated Regions
  • BCS1L protein, human
  • RNA, Messenger
  • ATPases Associated with Diverse Cellular Activities
  • Electron Transport Complex III

Associated data

  • GENBANK/EF672347