A new mutation in the C-SH2 domain of PTPN11 causes Noonan syndrome with multiple giant cell lesions

J Hum Genet. 2014 Jan;59(1):57-9. doi: 10.1038/jhg.2013.118. Epub 2013 Nov 14.

Abstract

Noonan syndrome (NS), an autosomal dominant multisystem disorder, is caused by the dysregulation of the RAS-MAPK pathway and is characterized by short stature, heart defects, pectus excavatum, webbed neck, learning problems, cryptorchidism and facial dysmorphism. We here present the clinical and molecular characterization of a family with NS and multiple giant cell lesions (MGCLs). The proband is a 12-year-old girl with NS and MGCL. Her mother shows typical NS without MGCL. Whole-exome sequencing of the girl, her mother and her healthy maternal grand parents revealed a previously unobserved mutation in exon 5 of the PTPN11 gene (c.598 A>T; p.N200Y), transmitted from the mother to the proband. As no other modification in the RAS-MAPK pathway genes as related to Rasopathies was detected in the proband, this report demonstrates for the first time that a unique mutation affecting this, otherwise unaffected signaling route, can cause both NS and NS/MGCL in the same family. This observation further confirms that NS/MGCL is not a distinct entity but rather that MGCL represents a rare complication of NS. Moreover, the localization of the p.N200Y mutation suggests an alternative molecular mechanism for the excessive phosphatase activity of the PTPN11-encoded protein.

Publication types

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

MeSH terms

  • Biopsy
  • Child
  • DNA Mutational Analysis
  • Exome
  • Facies
  • Female
  • Giant Cells / pathology*
  • Humans
  • Male
  • Mutation*
  • Noonan Syndrome / genetics*
  • Noonan Syndrome / pathology*
  • Phenotype
  • Protein Tyrosine Phosphatase, Non-Receptor Type 11 / chemistry
  • Protein Tyrosine Phosphatase, Non-Receptor Type 11 / genetics*
  • Synovial Membrane / metabolism
  • Synovial Membrane / pathology
  • src Homology Domains / genetics*

Substances

  • Protein Tyrosine Phosphatase, Non-Receptor Type 11