Intronic mutation in the PGK1 gene may cause recurrent myoglobinuria by aberrant splicing.

Shirakawa, Kentaro; Takahashi, Yoshitomo; Miyajima, Hiroaki · Neurology · 2006

case_report · Level V

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Abstract

A 33-year-old man with mental retardation and recurrent myoglobinuria demonstrated a deficiency in the phosphoglycerate kinase 1 (PGK1) activity of his muscles and erythrocytes. His PGK1 gene had intronic G-to-A substitution 5 nucleotides downstream from the normal exon 7 5' splice site (IVS7 + 5 G>A). This novel mutation results in a frame shift due to the insertion of 52 bp of intron 7 in the mature mRNA by aberrant splicing.

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