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. Results/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.
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