Phenotypical spectrum of cerebellar ataxia associated with a novel mutation in the CA8 gene, encoding carbonic anhydrase (CA) VIII

Am J Med Genet B Neuropsychiatr Genet. 2011 Dec;156B(7):826-34. doi: 10.1002/ajmg.b.31227. Epub 2011 Aug 2.

Abstract

We define the neurological characteristics of familial cases from multiple branches of a large consanguineous family with cerebellar ataxia, mental retardation (MR), and dysequilibrium syndrome type 3 caused by a mutation in the recently cloned CA8 gene. The linkage analysis revealed a high logarithm of the odds (LOD) score region on 8q that harbors the CA8 in which a novel homozygous c.484G>A (p.G162R) mutation was identified in all seven affected members. The patients had variable cerebellar ataxia and mild cognitive impairment without quadrupedal gait. The brain MRI showed variable cerebellar volume loss and ill-defined peritrigonal white matter abnormalities. The Fluorodeoxyglucose Positron Emission Tomography (FDG PET) revealed hypometabolic cerebellar hemispheres, temporal lobes, and mesial cortex. This report expands the neurological and radiological phenotype associated with CA8 mutations. CA8 involvement should be considered in the differential diagnosis of other genetically unresolved autosomal recessive cerebellar ataxias.

Publication types

  • Case Reports

MeSH terms

  • Adolescent
  • Amino Acid Sequence
  • Base Sequence
  • Biomarkers, Tumor / chemistry
  • Biomarkers, Tumor / genetics*
  • Brain / diagnostic imaging
  • Brain / pathology
  • Cerebellar Ataxia / diagnostic imaging
  • Cerebellar Ataxia / enzymology*
  • Cerebellar Ataxia / genetics*
  • Cerebellar Ataxia / pathology
  • Child
  • Child, Preschool
  • DNA Mutational Analysis
  • Female
  • Gene Regulatory Networks / genetics
  • Genetic Predisposition to Disease*
  • Humans
  • Infant
  • Infant, Newborn
  • Magnetic Resonance Imaging
  • Male
  • Molecular Sequence Data
  • Mutation / genetics*
  • Phenotype
  • Positron-Emission Tomography
  • Young Adult

Substances

  • Biomarkers, Tumor
  • CA8 protein, human