Published in

Wiley, Annals of Neurology, 1(59), p. 190-195, 2005

DOI: 10.1002/ana.20705

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COL6A1 genomic deletions in Bethlem myopathy and Ullrich muscular dystrophy

This paper is available in a repository.
This paper is available in a repository.

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Abstract

We have identified highly similar heterozygous COL6A1 genomic deletions, spanning from intron 8 to exon 13 or intron 13, in two patients with Ullrich congenital muscular dystrophy and the milder Bethlem myopathy. The 5' breakpoints of both deletions are located within a minisatellite in intron 8. The mutations cause in-frame deletions of 66 and 84 amino acids in the amino terminus of the triple-helical domain, leading to intracellular accumulation of mutant polypeptides and reduced extracellular collagen VI microfibrils. Our studies identify a deletion-prone region in COL6A1 and suggest that similar mutations can lead to congenital muscle disorders of different clinical severity.