A splice site mutation in the methyltransferase gene FTSJ1 in Xp11.23 is associated with non-syndromic mental retardation in a large Belgian family (MRX9)

J Med Genet. 2004 Sep;41(9):679-83. doi: 10.1136/jmg.2004.019000.

Abstract

Mental retardation is the most frequent cause of serious handicap in children and young adults. The underlying causes of this heterogeneous condition are both acquired and genetically based. A recently performed refinement of the linkage interval in a large Belgian family with mild to severe non-syndromic X linked mental retardation, classified as MRX9, revealed a candidate region of 11.3 Mb between markers DXS228 and DXS1204 on the short arm of the X chromosome. In order to identify the underlying disease gene in the MRX9 family, we established a gene catalogue for the candidate region and performed comprehensive mutation analysis by direct sequencing. A human homologue of the bacterial 23S rRNA methyltransferase Fstj, the FTSJ1 gene, is located within this region and displayed a sequence alteration in the conserved acceptor splice site of intron 3 (IVS3-2A>G) in all tested patients and carrier females of this family. In contrast, it was absent in all unaffected male family members tested. The mutation results in skipping of exon 4 and introduces a premature stop codon in exon 5, probably leading to a severely truncated protein. Our finding indicates that a protein, possibly associated with ribosomal stability, can be linked to X linked mental retardation (XLMR).

Publication types

  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Amino Acid Sequence
  • Belgium
  • Chromosomes, Human, X / genetics*
  • Female
  • Humans
  • Intellectual Disability / genetics*
  • Male
  • Methyltransferases / chemistry
  • Methyltransferases / genetics*
  • Molecular Sequence Data
  • Mutation / genetics*
  • Nuclear Proteins / chemistry
  • Nuclear Proteins / genetics*
  • Pedigree
  • RNA Splice Sites / genetics*
  • RNA, Messenger / analysis
  • RNA, Messenger / genetics

Substances

  • Nuclear Proteins
  • RNA Splice Sites
  • RNA, Messenger
  • FTSJ1 protein, human
  • Methyltransferases