Novel mutations in African American patients with glycogen storage disease Type II. Mutations in brief no. 209. Online

Hum Mutat. 1999;13(1):83-4. doi: 10.1002/(sici)1098-1004(1999)13:1<83::aid-humu13>3.0.co;2-2.

Abstract

The infantile form of GSD II (an inherited deficiency of the lysosomal enzyme, acid alpha-glucosidase, Pompe disease) is a severe and invariably fatal disease characterized by a rapidly progressive generalized hypotonia, hepatomegaly, and cardiomegaly. We have recently demonstrated that African American patients share a common nonsense R854X mutation in exon 18 (Becker et al., 1998). Two other mutations, D645E and M519V, have been identified in individual African American patients (Hermans et al., 1993a; Huie et al., 1994a). We describe here three novel mutations in this population group: a missense W481R in exon 10, a deletion of a T1441 in exon 10, and a splicing defect at the 5' donor site of intron 8 (IVS g+la) . The splicing defect is shared by two unrelated patients and it is linked to intragenic polymorphic sites identical to those found in patients bearing the common R854X mutation.

MeSH terms

  • Amino Acid Substitution / genetics
  • Black People / genetics*
  • Black or African American
  • Glycogen Storage Disease Type II / genetics*
  • Humans
  • Mutation / genetics*
  • Mutation, Missense / genetics