Evidence against RAB40AL being the locus for Martin-Probst X-linked deafness-intellectual disability syndrome

Hum Mutat. 2014 Oct;35(10):1171-4. doi: 10.1002/humu.22620. Epub 2014 Aug 7.

Abstract

RAB40AL has been reported as the locus for Martin-Probst syndrome (MPS), an X-linked deafness-intellectual disability syndrome. The report was based on segregation of a missense change p.D59G with the disease in a single family and in vitro localization studies. We found the p.D59G variant by whole-exome sequencing in two patients; however, the diagnosis of MPS was excluded in both cases. Furthermore, screening of control DNA samples (n = 810) from a general Polish population, using allele-specific PCR and direct DNA sequencing for verification, identified p.D59G in 8/405 males and 12/405 females. High prevalence of the p.D59G variant (2.47%) is typical for a common genetic variation observed in asymptomatic individuals. Our data question the role of RAB40AL mutation as a disease-causing change and the involvement of RAB40AL in MPS. Considering an increasing use of next-generation sequencing in the clinical setting, our finding is of practical diagnostic importance.

Keywords: Martin-Probst syndrome; RAB40AL; deafness; intellectual disability; nonpathogenic; whole-exome sequencing.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Abnormalities, Multiple / genetics*
  • Child
  • Female
  • Genetic Diseases, X-Linked / genetics*
  • Genetic Testing
  • Hearing Loss, Sensorineural / genetics*
  • Humans
  • Infant, Newborn
  • Intellectual Disability / genetics*
  • Male
  • Middle Aged
  • Mitochondrial Proteins / genetics*
  • Mutation, Missense*
  • Pedigree
  • Phenotype
  • Poland
  • Sequence Analysis, DNA
  • ras Proteins / genetics*

Substances

  • Mitochondrial Proteins
  • RAB40AL protein, human
  • ras Proteins

Supplementary concepts

  • Martin-Probst Deafness-Mental Retardation Syndrome