[Possible chemotherapy of muscular dystrophy caused by nonsense mutation].
Gentamicin, an aminoglycoside antibiotic 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 antibiotic with read-through activity in prokaryotes, restored dystrophin in skeletal and cardiac muscles of mdx mouse, an animal model for Duchenne type muscular dystrophy caused by nonsense 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.
Shiozuka
Masataka
M
Department of Life Sciences, Graduate School of Arts and Sciences, the University of Tokyo at Komaba.
Arakawa
Masayuki
M
Matsuda
Ryoichi
R
D000599:Amino Acids, Diamino / Q000627:therapeutic use*; D000818:Animals; D005839:Gentamicins / Q000627:therapeutic use; D006801:Humans; D009136:Muscular Dystrophies / Q000188:drug therapy* / Q000235:genetics; D014176:Protein Biosynthesis / Q000187:drug effects
D000599:Amino Acids, Diamino; D005839:Gentamicins; C100200:negamycin