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.
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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