GRID2 mutations span from congenital to mild adult-onset cerebellar ataxia
pmid: 25841024
GRID2 mutations span from congenital to mild adult-onset cerebellar ataxia
In a large family of Algerian origin, we aimed to identify the genetic mutation segregating with simultaneous presence of adult-onset, paucisymptomatic, slowly progressive, cerebellar ataxia in 7 adults and congenital ataxia in 1 child, and then to assess the involvement of GRID2 mutations in 144 patients with congenital cerebellar ataxia.We used a combined approach of linkage analysis and whole-exome sequencing in one family, and a targeted gene panel sequencing approach in 144 congenital ataxias.In the large family with spinocerebellar ataxia, we identified a missense mutation (c.1966C>G/p.Leu656Val) in the GRID2 gene, in a heterozygous state in adults, and in a homozygous state in one child with congenital ataxia, compatible with a semidominant transmission pattern. In 144 patients affected with congenital ataxia, we identified 2 missense de novo GRID2 mutations in 2 children (c.1960G>A/p.Ala654Thr, c.1961C>A/p.Ala654Asp). They affect the same amino acid as the previously described Lurcher mutation in mice; the variant in the large family concerns a nearby amino acid.In humans, GRID2 had only been involved in ataxia through complete loss-of-function mutations due to exon deletions. We report the first point mutations in this gene, with putative gain-of-function mechanisms, and a semidominant transmission as was observed in the Lurcher mice model. Of note, cerebellar ataxia is the core phenotype, but with variable severity ranging from very mild adult-onset to congenital-onset ataxias linked to both the heterozygous and homozygous state of the variant, and the position of the mutation.
- Medical Genetics & Functional Genomics France
- Université Catholique de Louvain Belgium
- Centre national de la recherche scientifique France
- Panthéon-Assas University France
- ICM Partners United States
Adult, Male, Adolescent, Cerebellar Ataxia, Genetic Linkage, Sequence Analysis, DNA, Middle Aged, Magnetic Resonance Imaging, [SDV.MHEP.CSC] Life Sciences [q-bio]/Human health and pathology/Cardiology and cardiovascular system, Pedigree, [SDV] Life Sciences [q-bio], Receptors, Glutamate, Algeria, Child, Preschool, Humans, Point Mutation, Exome, Female, Child, Aged
Adult, Male, Adolescent, Cerebellar Ataxia, Genetic Linkage, Sequence Analysis, DNA, Middle Aged, Magnetic Resonance Imaging, [SDV.MHEP.CSC] Life Sciences [q-bio]/Human health and pathology/Cardiology and cardiovascular system, Pedigree, [SDV] Life Sciences [q-bio], Receptors, Glutamate, Algeria, Child, Preschool, Humans, Point Mutation, Exome, Female, Child, Aged
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