Mental retardation and ataxia due to normotriglyceridemic hypobetalipoproteinemia

Ann Neurol. 2005 Jul;58(1):160-3. doi: 10.1002/ana.20531.

Abstract

A 12-year-old boy with mental retardation, obesity, ataxia, and visual impairment was shown to have normal fasting plasma triglyceride but low cholesterol and vitamin E levels. Investigations indicated that he was compound heterozygous for two mutations in the apolipoprotein B gene (APOB), resulting in a failure to express apolipoprotein B-100, yet retain apolipoprotein B-48 production. The proband therefore was able to form chylomicrons, but not a low-density lipoprotein capable of receptor-mediated endocytosis. This resulted in chronic vitamin E deficiency. We suggest the term normotriglyceridemic hypobetalipoproteinemia for this easily recognizable condition.

Publication types

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

MeSH terms

  • Apolipoproteins B / blood
  • Apolipoproteins B / genetics*
  • Ataxia / etiology*
  • Blotting, Western
  • Child
  • Child, Preschool
  • Cholesterol, LDL / blood
  • Chylomicrons
  • Humans
  • Hypobetalipoproteinemias / complications*
  • Hypobetalipoproteinemias / genetics*
  • Infant
  • Intellectual Disability / etiology*
  • Male
  • Mutation
  • Obesity / etiology
  • Polymerase Chain Reaction
  • Triglycerides / blood
  • Vitamin E / blood

Substances

  • Apolipoproteins B
  • Cholesterol, LDL
  • Chylomicrons
  • Triglycerides
  • Vitamin E