title: FXTAS: a bad RNA and a hope for a cure.
abstract: BACKGROUND
  Fragile X-associated tremor/ataxia syndrome (FXTAS) is a newly identified neurodegenerative disorder due to intermediate expansion of trinucleotide CGG repeats (55 - 200 repeats) in the 5' untranslated region (UTR) of the Fragile X mental retardation 1 (FMR1) gene. FXTAS is now considered to be one of the most common inherited neurodegenerative disorders in males.
  
  
  OBJECTIVE
  To examine the future of potential therapies for this late-onset disease.
  
  
  METHODS
  Examination of relevent literature.
  
  
  CONCLUSIONS
  Accumulating evidence indicates that overproduced riboCGG repeats in the 5' UTR of FMR1 mRNA are toxic. Recently, proteins that bind specifically to rCGG repeats were identified. Progress in understanding the molecular pathogenesis of FXTAS, plus the availability of different animal models are discussed.
authors.affiliation: Emory University School of Medicine, Department of Human Genetics, Atlanta, GA 30322, USA.
authors.forename: Ge
authors.initials: G
authors.lastname: Shan
authors.forename: Shunliang
authors.initials: S
authors.lastname: Xu
authors.forename: Peng
authors.initials: P
authors.lastname: Jin
mesh_terms: D000818:Animals; D001259:Ataxia / Q000175:diagnosis / Q000235:genetics* / Q000628:therapy; D051860:Fragile X Messenger Ribonucleoprotein 1 / Q000737:chemistry / Q000235:genetics*; D015316:Genetic Therapy / Q000639:trends; D006801:Humans; D012313:RNA / Q000737:chemistry / Q000235:genetics*; D013577:Syndrome; D014202:Tremor / Q000175:diagnosis / Q000235:genetics* / Q000628:therapy
chemical_list: D051860:Fragile X Messenger Ribonucleoprotein 1; D012313:RNA; C497683:FMR1 protein, human