A 5' splice region G-->C mutation in exon 3 of the human beta-spectrin gene leads to decreased levels of beta-spectrin mRNA and is responsible for dominant hereditary spherocytosis (spectrin Guemene-Penfao)

Br J Haematol. 1998 Jan;100(1):90-8. doi: 10.1046/j.1365-2141.1998.00530.x.

Abstract

We studied a family with autosomal dominant hereditary spherocytosis (HS) associated with a mild spectrin deficiency. Linkage analysis using two microsatellite markers (D14S63 and D14S271) very close to the beta-spectrin gene (SPTB) showed that HS co-segregated with alleles of these microsatellite markers and the linkage between the marker and HS was statistically significant. The presence of a beta-spectrin protein polymorphism (beta-spectrin Vay; A1880V) in trans of the HS allele was not itself deleterious, but allowed the detection of decreased membrane expression of the spherocytic beta-spectrin allele in two HS-affected subjects. Direct sequencing of the coding exons of the beta-spectrin gene in one affected subject showed the presence of a G-->C transversion at the terminal nucleotide of exon 3, which did not change the leucine codon 100 (CTG-->CTC). The presence of the mutation was confirmed by restriction enzyme digestion at the DNA level in all affected SH members of the family. The G-->C mutation severely reduced the utilization of the 5' splice site and resulted in aberrant mRNA splicing with intron 3 retention.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Base Sequence
  • Exons / genetics
  • Female
  • Humans
  • Male
  • Molecular Sequence Data
  • Mutation*
  • Pedigree
  • Polymerase Chain Reaction
  • RNA Splicing / genetics*
  • RNA, Messenger / genetics
  • Spectrin / genetics*
  • Spherocytosis, Hereditary / genetics*

Substances

  • RNA, Messenger
  • Spectrin

Associated data

  • GENBANK/AF013172
  • GENBANK/AF013173
  • GENBANK/AF013174
  • GENBANK/AF013175
  • GENBANK/AF013176
  • GENBANK/AF013177
  • GENBANK/AF013178
  • GENBANK/AF013179
  • GENBANK/AF013180
  • GENBANK/AF013181
  • GENBANK/AF013182
  • GENBANK/AF013183
  • GENBANK/AF013184
  • GENBANK/AF013185
  • GENBANK/AF013186
  • GENBANK/AF013187