In vitro readthrough of termination codons by gentamycin in the Stüve-Wiedemann Syndrome

Eur J Hum Genet. 2010 Jan;18(1):130-2. doi: 10.1038/ejhg.2009.122.

Abstract

The Stüve-Wiedemann Syndrome (SWS) is a frequently lethal chondrodysplasia caused by null mutations in the leukemia inhibitory factor receptor gene (LIFR) responsible for an impaired activation of the JAK-STAT pathway after LIF stimulation. Most LIFR mutations are nonsense mutations, thus prompting us to investigate the impact of aminoglycosides on the readthrough of premature termination codons (PTCs). Culturing skin fibroblasts from three SWS patients and controls for 48 h in the presence of gentamycin (200-500 microg/ml) partially restored the JAK-STAT3 pathway when stimulated by LIF. Consistently, quantitative RT-PCR analysis showed that gentamycin stabilized LIFR mRNAs carrying UGA premature termination codons. We conclude that high gentamycin concentrations can partially restore functional LIFR protein synthesis in vitro, prompting us to investigate PTC readthrough using less toxic and more efficient drugs in this presently untreatable lethal condition.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Abnormalities, Multiple / enzymology
  • Abnormalities, Multiple / genetics*
  • Codon, Terminator / genetics*
  • Enzyme Activation / drug effects
  • Fibroblasts / drug effects
  • Fibroblasts / metabolism
  • Gentamicins / pharmacology*
  • Humans
  • Janus Kinases / metabolism
  • Leukemia Inhibitory Factor / pharmacology
  • Leukemia Inhibitory Factor Receptor alpha Subunit / genetics
  • STAT Transcription Factors / metabolism
  • Signal Transduction / drug effects
  • Syndrome

Substances

  • Codon, Terminator
  • Gentamicins
  • LIFR protein, human
  • Leukemia Inhibitory Factor
  • Leukemia Inhibitory Factor Receptor alpha Subunit
  • STAT Transcription Factors
  • Janus Kinases