Abstract
Detailed clinical, neuroradiological, histological, biochemical, and genetic investigations were undertaken in a child suffering from Leigh syndrome. The clinical symptoms started at age five months and led to a severe progressive neurodegenerative disorder causing epilepsy, psychomotor retardation, and tetraspasticity. Biochemical measurement of skeletal muscle showed a severe decrease in mitochondrial complex II. Sequencing of SDHA revealed compound heterozygosity for a nonsense mutation in exon 4 (W119X) and a missense mutation in exon 3 (A83V), both absent in normal controls. In six additional patients--five with Leigh or Leigh-like syndrome and one with neuropathy and ataxia associated with isolated deficiency of complex II--mutations in SDHA were not detected, indicating genetic heterogeneity.
MeSH Terms
Atrophy/pathology
Biopsy
Brain/pathology
Cerebral Cortex/pathology
Child
Child, Preschool
DNA/analysis
DNA, Complementary/analysis
Disease Progression
Exons/genetics
Female
Flavoproteins/genetics
Functional Laterality
Humans
Immunoblotting
Infant
Leigh Disease/diagnosis,genetics
Magnetic Resonance Imaging
Male
Muscle, Skeletal/pathology
Point Mutation/genetics
Polymerase Chain Reaction
Protein Subunits/genetics
RNA/analysis
Succinate Dehydrogenase/genetics
Chemicals
DNA, Complementary
Flavoproteins
Protein Subunits
RNA
DNA
Succinate Dehydrogenase
Authors & Affiliations
9 authors, click to expand affiliations / ORCID
Horváth R
Metabolic Disease Centre Munich-Schwabing, Institute of Clinical Chemistry, Academic Hospital Schwabing, Kölner Platz 1, 80804 Munich, Germany. Rita.Horvath@lrz.uni-muenchen.de
Abicht A
Holinski-Feder E
Laner A
Gempel K
Prokisch H
Lochmüller H
Klopstock T
Jaksch M
References (10)
10 references, click to expand
-
Compound heterozygous mutations in the flavoprotein gene of the respiratory chain complex II in a patient with Leigh syndrome.
Hum Genet. 2000 Feb;106(2):236-43
PMID: 10746566
-
Late-onset optic atrophy, ataxia, and myopathy associated with a mutation of a complex II gene.
Ann Neurol. 2000 Sep;48(3):330-5
PMID: 10976639
-
DHPLC mutation analysis of the hereditary nonpolyposis colon cancer (HNPCC) genes hMLH1 and hMSH2.
J Biochem Biophys Methods. 2001 Jan 30;47(1-2):21-32
PMID: 11179758
-
Inborn errors of complex II--unusual human mitochondrial diseases.
Biochim Biophys Acta. 2002 Jan 17;1553(1-2):117-22
PMID: 11803021
-
Investigation of the role of SDHB inactivation in sporadic phaeochromocytoma and neuroblastoma.
Br J Cancer. 2004 Nov 15;91(10):1835-41
PMID: 15505628
-
Homozygous Gly555Glu mutation in the nuclear-encoded 70 kDa flavoprotein gene causes instability of the respiratory chain complex II.
Am J Med Genet A. 2003 Jul 1;120A(1):13-8
PMID: 12794685
-
A mitochondrial encephalomyopathy: the first case with an established defect at the level of coenzyme Q.
Eur J Pediatr. 1986 Feb;144(5):441-4
PMID: 3956532
-
Mutation of a nuclear succinate dehydrogenase gene results in mitochondrial respiratory chain deficiency.
Nat Genet. 1995 Oct;11(2):144-9
PMID: 7550341
-
Deficiency of complex II of the mitochondrial respiratory chain in late-onset optic atrophy and ataxia.
Ann Neurol. 1996 Feb;39(2):224-32
PMID: 8967754
-
Succinate dehydrogenase and human diseases: new insights into a well-known enzyme.
Eur J Hum Genet. 2002 May;10(5):289-91
PMID: 12082502