Variable Expressivity of Wolfram Syndrome in a Family with Multiple Affected Subjects.

Purpose: To study the genetic basis and clinical manifestations of Wolfram syndrome in a multi-affected family. Methods: Complete clinical examinations including urological, ophthalmic, neurological, and endocrinologic assessment were performed for three affected family members. Genomic DNA was extr...

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Publicado en:Journal of Ophthalmic & Vision Research Vol. 16; no. 4; pp. 602 - 611
Autores principales: Mirrahimi, Mehraban, Safi, Sare, Mohammadzadeh, Maryam, Doozandeh, Azadeh, Suri, Fatemeh
Formato: case study diagnostic images pictorial tables/charts Journal Article
Publicado: Knowledge E DMCC Oct-Dec2021
Acceso en línea:Ver este registro en EBSCOhost
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      dt: Oct-Dec2021
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        atl: Variable Expressivity of Wolfram Syndrome in a Family with Multiple Affected Subjects.
      aug:
        au:
          Mirrahimi, Mehraban
          Safi, Sare
          Mohammadzadeh, Maryam
          Doozandeh, Azadeh
          Suri, Fatemeh
        affil: Ocular Tissue Engineering Research Center, Research Institute for Ophthalmology and Vision Science, Shahid Beheshti University of Medical Sciences, Tehran, Iran
      sug:
        subj:
          Genomics
          Wolfram Syndrome
          Affective Symptoms
          DNA
      ab: Purpose: To study the genetic basis and clinical manifestations of Wolfram syndrome in a multi-affected family. Methods: Complete clinical examinations including urological, ophthalmic, neurological, and endocrinologic assessment were performed for three affected family members. Genomic DNA was extracted from peripheral blood leukocytes with salting out method and all WFS1 exons and their flanking regions were sequenced. Candidate variation was screened for segregation in the pedigree by Sanger sequencing. Results: A known pathogenic missense mutation in WFS1 gene (c.1885C>T which leads to p.Arg629Trp in the encoded protein) was identified in all affected individuals. Both clinical and genetic investigations confirmed Wolfram syndrome diagnosis with variable phenotypic features. Conclusion: Identical mutations in the Wolfram syndrome causative gene can lead to variable manifestations of the syndrome even in the same family. Although the medical findings and clinical examination are imperative for the diagnosis of Wolfram syndrome, genetic testing is useful to confirm the diagnosis, especially in cases with possible reduced penetrance of the characteristic signs.
      pubtype: Academic Journal
      doctype:
        case study
        diagnostic images
        pictorial
        tables/charts
        Journal Article
      ougenre: Article
    language: English
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