Myoclonus-dystonia due to genomic deletions in the epsilon-sarcoglycan gene.

Asmus, Friedrich; Salih, Farid; Hjermind, Lena Elisabeth; Ostergaard, Karen; Munz, Marita; Kühn, Andrea A; Dupont, Erik; Kupsch, Andreas et al. · Ann Neurol · 2005

case_control · Level III

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Abstract

Direct genomic DNA sequencing fails to detect epsilon-sarcoglycan (SGCE) mutations in up to 30% of familial myoclonus-dystonia (M-D) cases. We identified novel large heterozygous deletions of SGCE exon 5 or exon 6 in two M-D pedigrees. Like nonsense mutations, exon rearrangements result in the generation of premature stop codons downstream of the deleted exon. SGCE exon dosage assays may identify additional families with SGCE mutation and thus reduce "genetic heterogeneity."

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