Abnormal processing of the glycoprotein IIb transcript due to a nonsense mutation in exon 17 associated with Glanzmann's thrombasthenia

Thromb Haemost. 1995 May;73(5):756-62.

Abstract

We analyzed the molecular genetic defect responsible for type I Glanzmann's thrombasthenia in a Japanese patient. In an immunoblot assay using polyclonal anti-GpIIb-IIIa antibodies, some GPIIIa (15% of normal amount) could be detected in the patient's platelets, whereas GPIIb could not (< 2% of normal amount). Nucleotide sequence analysis of platelet GPIIb mRNA-derived polymerase chain reaction (PCR) products revealed that patient's GPIIb cDNA had a 75-bp deletion in the 3' boundary of exon 17 resulting in an in-frame deletion of 25 amino acids. DNA analysis and family study revealed that the patient was a compound heterozygote of two GPIIb gene defects. One allele derived from her father was not expressed in platelets, and the other allele derived from her mother had a 9644C--> T mutation which was located at the position -3 of the splice donor junction of exon 17 and resulted in a termination codon (TGA). Moreover, quantitative analysis demonstrated that the amount of the abnormal GPIIb transcript in the patient's platelets was markedly reduced. Thus, the C --> T mutation resulting in the abnormal splicing of GPIIb transcript and the reduction in its amount is responsible for Glanzmann's thrombasthenia.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Amino Acid Sequence
  • Base Sequence
  • Deoxyribonucleases, Type II Site-Specific
  • Exons / genetics
  • Female
  • Genes
  • Heterozygote
  • Humans
  • Japan
  • Molecular Sequence Data
  • Platelet Glycoprotein GPIIb-IIIa Complex / genetics*
  • Platelet Glycoprotein GPIIb-IIIa Complex / metabolism
  • Polymerase Chain Reaction
  • Polymorphism, Restriction Fragment Length
  • RNA Processing, Post-Transcriptional*
  • RNA, Messenger / genetics*
  • RNA, Messenger / metabolism
  • Sequence Deletion*
  • Thrombasthenia / classification
  • Thrombasthenia / genetics*

Substances

  • Platelet Glycoprotein GPIIb-IIIa Complex
  • RNA, Messenger
  • Deoxyribonucleases, Type II Site-Specific
  • TCGA-specific type II deoxyribonucleases