Differential usage of transcriptional start sites and polyadenylation sites in FMR1 premutation alleles

Nucleic Acids Res. 2011 Aug;39(14):6172-85. doi: 10.1093/nar/gkr100. Epub 2011 Apr 7.

Abstract

5'- and 3'-untranslated regions (UTRs) are important regulators of gene expression and play key roles in disease progression and susceptibility. The 5'-UTR of the fragile X mental retardation 1 (FMR1) gene contains a CGG repeat element that is expanded (>200 CGG repeats; full mutation) and methylated in fragile X syndrome (FXS), the most common form of inherited intellectual disability (ID) and known cause of autism. Significant phenotypic involvement has also emerged in some individuals with the premutation (55-200 CGG repeats), including fragile X-associated premature ovarian insufficiency (FXPOI) in females, and the neurodegenerative disorder, fragile X-associated tremor/ataxia syndrome (FXTAS), in older adult carriers. Here, we show that FMR1 mRNA in human and mouse brain is expressed as a combination of multiple isoforms that use alternative transcriptional start sites and different polyadenylation sites. Furthermore, we have identified a novel human transcription start site used in brain but not in lymphoblastoid cells, and have detected FMR1 isoforms generated through the use of both canonical and non-canonical polyadenylation signals. Importantly, in both human and mouse, a specific regulation of the UTRs is observed in brain of FMR1 premutation alleles, suggesting that the transcript variants may play a role in premutation-related pathologies.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • 3' Untranslated Regions
  • 5' Untranslated Regions
  • Alleles*
  • Animals
  • Base Sequence
  • Brain / metabolism
  • Fragile X Mental Retardation Protein / genetics*
  • Fragile X Mental Retardation Protein / metabolism
  • Humans
  • Mice
  • Molecular Sequence Data
  • Mutation
  • Polyadenylation*
  • Transcription Initiation Site*
  • Trinucleotide Repeats
  • Untranslated Regions*

Substances

  • 3' Untranslated Regions
  • 5' Untranslated Regions
  • FMR1 protein, human
  • Fmr1 protein, mouse
  • Untranslated Regions
  • Fragile X Mental Retardation Protein