AIMP1 deficiency presents as a cortical neurodegenerative disease with infantile onset

Neurogenetics. 2014 Aug;15(3):157-9. doi: 10.1007/s10048-014-0411-3. Epub 2014 Jun 24.

Abstract

We report the second family with AIMP1 deficiency, due to a homozygous truncating AIMP1 (g.107248613 C > T) mutation. This female showed early-onset developmental arrest, intractable epileptic spasms, microcephaly, and a rapid clinical course leading to premature death, associated with cerebral atrophy and myelin deficiency on brain MRI. Clinical and neuroimaging findings are consistent with a primary neuronal degenerative disorder, rather than with the previously reported Perlizaeus-Merzbacher-like phenotype. Given its critical role in neurofilament assembly 16, impaired myelin formation is due to neuronal/axonal dysfunction. We propose that AIMP1 deficiency be added to the differential diagnosis of infantile onset, progressive neurodegenerative disease.

Publication types

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

MeSH terms

  • Age Factors
  • Brain / pathology
  • Cytokines / deficiency*
  • Cytokines / genetics*
  • Female
  • Humans
  • Infant, Newborn
  • Mutation
  • Neoplasm Proteins / deficiency*
  • Neoplasm Proteins / genetics*
  • Neurodegenerative Diseases / diagnosis*
  • Neurodegenerative Diseases / genetics*
  • RNA-Binding Proteins / genetics*
  • White Matter / pathology

Substances

  • Cytokines
  • Neoplasm Proteins
  • RNA-Binding Proteins
  • small inducible cytokine subfamily E, member 1