[Possible chemotherapy of muscular dystrophy caused by nonsense mutation].

Abstract

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.

• Authors: Masataka Shiozuka, Masayuki Arakawa, Ryoichi Matsuda
• Journal: Rinsho shinkeigaku = Clinical neurology (Rinsho Shinkeigaku) Vol. 44 Issue 11 Pg. 908-10 (Nov 2004) ISSN: 0009-918X [Print] Japan
• PMID: 15651328 (Publication Type: Journal Article, Review)

Chemical References

• Amino Acids, Diamino
• Gentamicins
• negamycin

Topics

• Amino Acids, Diamino (therapeutic use)
• Animals
• Gentamicins (therapeutic use)
• Humans
• Muscular Dystrophies (drug therapy, genetics)
• Protein Biosynthesis (drug effects)