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FXTAS: a progressive neurologic syndrome associated with Fragile X premutation.
Willemsen, Rob; Mientjes, Edwin; Oostra, Ben A.
Afiliación
  • Willemsen R; Department of Clinical Genetics, Erasmus MC, PO Box 1738, 3000 DR, Rotterdam, The Netherlands. r.willemsen@erasmusmc.nl
Curr Neurol Neurosci Rep ; 5(5): 405-10, 2005 Sep.
Article en En | MEDLINE | ID: mdl-16131424
The FMR1 gene is involved in two different syndromes: Fragile X syndrome and Fragile X-associated tremor/ataxia syndrome (FXTAS). Fragile X syndrome is a childhood disease and is associated with mental retardation as the main clinical characteristic, whereas FXTAS develops in men and women over 50 years of age. FXTAS represents a new form of inclusion disorder with a high prevalence in the general population. The neurologic phenotype of FXTAS includes intention tremor and ataxia. Associated features are dementia, parkinsonism, neuropathy, and autonomic dysfunction. Elevated FMR1 transcripts have been proposed as the molecular basis of the pathogenic mechanism leading to FXTAS. This review discusses recent developments in the clinical phenotype, prevalence and screening, animal models, and molecular mechanisms of RNA-based pathogenesis in FXTAS.
Asunto(s)
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Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Cromosomas Humanos X / Síndrome del Cromosoma X Frágil / Mutación Tipo de estudio: Etiology_studies / Prognostic_studies / Risk_factors_studies Límite: Humans Idioma: En Revista: Curr Neurol Neurosci Rep Asunto de la revista: NEUROLOGIA Año: 2005 Tipo del documento: Article País de afiliación: Países Bajos Pais de publicación: Estados Unidos
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Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Cromosomas Humanos X / Síndrome del Cromosoma X Frágil / Mutación Tipo de estudio: Etiology_studies / Prognostic_studies / Risk_factors_studies Límite: Humans Idioma: En Revista: Curr Neurol Neurosci Rep Asunto de la revista: NEUROLOGIA Año: 2005 Tipo del documento: Article País de afiliación: Países Bajos Pais de publicación: Estados Unidos