Detection of 28 novel mutations in the Wiskott-Aldrich syndrome and X-linked thrombocytopenia based on multiplex PCR

Blood Cells Mol Dis. 2007 Jul-Aug;39(1):102-6. doi: 10.1016/j.bcmd.2007.02.007. Epub 2007 Apr 2.

Abstract

The Wiskott-Aldrich syndrome (WAS) is an X-linked disorder including microthrombocytopenia, eczema and immunodeficiency. A mild form is known as the X-linked thrombocytopenia (XLT). We screened 150 individuals or families based on a multiplex PCR method. We found 28 novel mutations (7 missense, 1 nonsense, 1 nonstop change, 5 splice site mutations and 14 deletions or insertions). The method relied on the co-synthesis of 5 amplicons and direct sequencing, optimizing the novel protocol proposed by Jones et al. [L.N. Jones, M.I. Lutskiy, J. Cooley, et al. A novel protocol to identify mutations in patients with Wiskott-Aldrich syndrome, Blood Cells Mol. Dis. 28 (2002) 392-398]. It was thus possible to identify faster and at a lower cost the mutations in newly diagnosed patients. The mutation distribution, according to the type, was in keeping with the distribution reported previously. No clear-cut genotype-phenotype correlation was observed.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • DNA Mutational Analysis
  • Diagnosis, Differential
  • Female
  • France
  • Genetic Diseases, X-Linked / diagnosis
  • Genetic Diseases, X-Linked / genetics*
  • Genotype
  • Humans
  • Male
  • Mutation*
  • Polymerase Chain Reaction*
  • Thrombocytopenia / diagnosis
  • Thrombocytopenia / genetics*
  • Wiskott-Aldrich Syndrome / diagnosis
  • Wiskott-Aldrich Syndrome / genetics*
  • Wiskott-Aldrich Syndrome Protein / genetics*

Substances

  • WAS protein, human
  • Wiskott-Aldrich Syndrome Protein