Ataxia and pancytopenia caused by a mutation in TINF2

Hum Genet. 2008 Dec;124(5):507-13. doi: 10.1007/s00439-008-0576-7. Epub 2008 Nov 1.

Abstract

The syndrome of ataxia-pancytopenia is an autosomal dominant disorder characterized by cerebellar ataxia, peripheral neuropathies, pancytopenia and a predilection to myelodysplastic syndrome and acute myeloid leukemia. The genetic basis of this condition is unknown. We describe a child who presented with ataxia and pancytopenia and was found to have a heterozygous mutation, c.845G>A (Arg282His) in TINF2, a gene recently reported to be mutated in a subset of patients with autosomal dominant dyskeratosis congenita. We propose that some cases of ataxia-pancytopenia may be affected by DC.

Publication types

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

MeSH terms

  • Adult
  • Amino Acid Substitution
  • Base Sequence
  • Cerebellar Ataxia / genetics*
  • DNA / genetics
  • Dyskeratosis Congenita / genetics
  • Female
  • Genes, Dominant
  • Humans
  • Infant
  • Male
  • Pancytopenia / genetics*
  • Point Mutation*
  • Syndrome
  • Telomere / genetics
  • Telomere-Binding Proteins / genetics*

Substances

  • TINF2 protein, human
  • Telomere-Binding Proteins
  • DNA