Study of the molecular defects in pyruvate kinase deficient patients affected by nonspherocytic hemolytic anemia

Blood Cells Mol Dis. 1995;21(1):49-55. doi: 10.1006/bcmd.1995.0008.

Abstract

We have examined DNA from fifteen unrelated pyruvate kinase deficient patients with hereditary nonspherocytic hemolytic anemia (HNSHA) for the molecular alterations responsible for the enzyme deficiency. All but 3 of the 30 putative mutations were identified. Fourteen different mutations were found. Nine were missense mutations: 320 T-->C, 823 G-->C, 1276 C-->T, 1378 G-->A, 1484 C-->T, 1529 G-->A, 1654 G-->A, 1675 C-->G; three were nonsense mutations: 603 G-->A, 721 G-->T, 1501 C-->T; one was an insertion at 1574 GGG-->GGGG and the other a three nucleotide in-frame deletion 391-392-393 ATC. Eight of these mutations have not been previously described. We also investigated all of the patients for the C/A polymorphism at nt 1705 and the microsatellite ATT repeat in intron 11. All of the mutations that had previously been reported by us (391-393del, 721T, 1484T, 1529A) were found in the context of the same haplotype as the earlier cases, supporting the concept that each may have a single origin.

Publication types

  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Anemia, Hemolytic, Congenital Nonspherocytic / enzymology
  • Anemia, Hemolytic, Congenital Nonspherocytic / ethnology
  • Anemia, Hemolytic, Congenital Nonspherocytic / genetics*
  • Base Sequence
  • DNA Mutational Analysis
  • DNA, Satellite / genetics
  • Ethnicity / genetics
  • Female
  • Haplotypes / genetics
  • Humans
  • Isoenzymes / deficiency
  • Isoenzymes / genetics*
  • Male
  • Molecular Sequence Data
  • Mutation*
  • Pedigree
  • Polymorphism, Genetic
  • Polymorphism, Single-Stranded Conformational
  • Promoter Regions, Genetic
  • Pyruvate Kinase / deficiency
  • Pyruvate Kinase / genetics*
  • Repetitive Sequences, Nucleic Acid

Substances

  • DNA, Satellite
  • Isoenzymes
  • Pyruvate Kinase