Gene/Protein Disease Symptom Drug Enzyme Compound
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Query: UMLS:C0026850 (muscular dystrophy)
 5,870 document(s) hit in 31,850,051 MEDLINE articles (0.00 seconds)

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.
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PMID:[Possible chemotherapy of muscular dystrophy caused by nonsense mutation]. 1565 28