PRRT2 mutations: exploring the phenotypical boundaries.

Background: Mutations in the proline-rich transmembrane protein 2 (PRRT2) gene have been identified in patients with benign (familial) infantile convulsions (B(F)IC), infantile convulsions with choreoathetosis (ICCA) and paroxysmal dyskinesias (PDs). However it remains unknown whether PRRT2 mutation...

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Publicado en:Journal of Neurology, Neurosurgery & Psychiatry Vol. 85; no. 4; pp. 462 - 466
Autores principales: Djémié, Tania, Weckhuysen, Sarah, Holmgren, Philip, Hardies, Katia, Van Dyck, Tine, Hendrickx, Rik, Schoonjans, An-Sofie, Van Paesschen, Wim, Jansen, Anna C, De Meirleir, Linda, Selim, Laila Abdel Moteleb, Girgis, Marian Y, Buyse, Gunnar, Lagae, Lieven, Smets, Katrien, Smouts, Iris, Claeys, Kristl G, Van den Bergh, Vic, Grisar, Thierry, Blatt, Ilan
Formato: research Journal Article
Publicado: BMJ Publishing Group Apr2014
Acceso en línea:Ver este registro en EBSCOhost
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      dt: Apr2014
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        atl: PRRT2 mutations: exploring the phenotypical boundaries.
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        au:
          Djémié, Tania
          Weckhuysen, Sarah
          Holmgren, Philip
          Hardies, Katia
          Van Dyck, Tine
          Hendrickx, Rik
          Schoonjans, An-Sofie
          Van Paesschen, Wim
          Jansen, Anna C
          De Meirleir, Linda
          Selim, Laila Abdel Moteleb
          Girgis, Marian Y
          Buyse, Gunnar
          Lagae, Lieven
          Smets, Katrien
          Smouts, Iris
          Claeys, Kristl G
          Van den Bergh, Vic
          Grisar, Thierry
          Blatt, Ilan
      sug:
        subj:
          Epilepsy
          Membrane Proteins
          Nerve Tissue Proteins
          Mutation
          Epilepsy Complications
          Epilepsy Diagnosis
          Female
          Learning Disorders Complications
          Learning Disorders
          Male
          Motor Skills Disorders Complications
          Motor Skills Disorders
          Pedigree
          Phenotype
          Female
          Male
      ab: Background: Mutations in the proline-rich transmembrane protein 2 (PRRT2) gene have been identified in patients with benign (familial) infantile convulsions (B(F)IC), infantile convulsions with choreoathetosis (ICCA) and paroxysmal dyskinesias (PDs). However it remains unknown whether PRRT2 mutations are causal in other epilepsy syndromes. After we discovered a PRRT2 mutation in a large family with ICCA containing one individual with febrile seizures (FS) and one individual with West syndrome, we analysed PRRT2 in a heterogeneous cohort of patients with different types of infantile epilepsy.Methods: We screened a cohort of 460 patients with B(F)IC or ICCA, fever related seizures or infantile epileptic encephalopathies. All patients were tested for point mutations using direct sequencing.Results: We identified heterozygous mutations in 16 individuals: 10 familial and 6 sporadic cases. All patients were diagnosed with B(F)IC, ICCA or PD. We were not able to detect mutations in any of the other epilepsy syndromes. Several mutation carriers had learning disabilities and/or impaired fine motor skills later in life.Conclusions: PRRT2 mutations do not seem to be involved in the aetiology of FS or infantile epileptic encephalopathies. Therefore B(F)IC, ICCA and PD remain the core phenotypes associated with PRRT2 mutations. The presence of learning disabilities or neuropsychiatric problems in several mutation carriers calls for additional clinical studies addressing this developmental aspect in more detail.
      pubtype: Academic Journal
      doctype:
        research
        Journal Article
      ougenre: Article
    language: English
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