Possible chemotherapy of muscular dystrophy caused by nonsense mutation

Gentamicin, an aminoglycoside antibiotics which causes read-through of premature termination codon during translation, has been used to rescue genetic diseases caused by nonsense mutation. Its strong side effects, however, has always threaten patients. In order to utilize other antibiotics with less side effects than gentamicin, we have shown that negamycin, a dipeptide antibiotics with read-through activity in prokaryotes, restored dystrophin in skeletal and cardiac muscles of mdx mouse, an animalumodel for Duchenne type muscular dystrophy caused by nonsens mutation. To avoid miscoding and emerging resistant bacteria for these read-through antibiotics, further drug design and high throughput screening of gentamicin- or negamycin-related molecules will be needed.