Molecular characterization of novel splice site mutation causing protein C deficiency

Blood Coagul Fibrinolysis. 2016 Jul;27(5):585-8. doi: 10.1097/MBC.0000000000000490.

Abstract

Congenital protein C deficiency is an inherited coagulation disorder associated with an elevated risk of venous thromboembolism. A Saudi Arabian male from a consanguineous family was admitted to neonatal intensive care unit in his first days of life because of transient tachypnea and hematuria. Laboratory investigations determined low platelet and protein C deficiency. Direct sequencing of PROC gene and RNA analysis were performed. Analysis of factor V Leiden (G1691A) and factor II (G20210A) mutations was also done. Novel homozygous splice site mutation c.796+3A>T was detected in the index case and segregation was confirmed in the family. RNA analysis revealed the pathogenicity of the mutation by skipping exon 8 of PROC gene and changing the donor splice site of the exon. Detection of the molecular cause of protein C deficiency reduces life threatening and facilitates inductive carrier testing, prenatal and preimplantation genetic diagnosis for families.

Publication types

  • Case Reports

MeSH terms

  • Base Sequence
  • Blood Platelets / metabolism
  • Blood Platelets / pathology
  • Consanguinity
  • Exons
  • Factor V / genetics
  • Gene Expression
  • Hematuria / blood
  • Hematuria / congenital
  • Hematuria / genetics*
  • Homozygote
  • Humans
  • Infant, Newborn
  • Introns
  • Male
  • Mutation*
  • Pedigree
  • Platelet Count
  • Protein C / genetics*
  • Protein C Deficiency / blood
  • Protein C Deficiency / congenital
  • Protein C Deficiency / genetics*
  • Prothrombin / genetics
  • RNA Splice Sites*
  • Saudi-Arabien
  • Tachypnea / blood
  • Tachypnea / congenital
  • Tachypnea / genetics*

Substances

  • Protein C
  • RNA Splice Sites
  • factor V Leiden
  • Factor V
  • Prothrombin