Treatment of hereditary diseases with gentamicin
US6475993B2 · kind B2 · utility
3Cited by
0References
1Claims
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Assignee
Inventor
Key dates
| Filing date | Dec 22, 1999 |
| Grant date | Nov 5, 2002 |
| Priority date | — |
| Expiry date | Dec 22, 2019 |
Classification
- Technology area (CPC A)Human Necessities
- CPC primaryA61K31/7036
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
This invention relates to a method of treating an inherited disease due to a point mutation producing a stop codon by administering an effective dose of an aminoglycoside antibiotic or a derivative thereof. Mdx mouse, which is an animal model for Duchenne muscular dystrophy, has been successfully treated with intramuscularly administered 1 and 5 mg gentamicin, which had for effect to suppress the premature stop mutation by inserting an amino acid at the stop codon. Dystrophin positive muscle fibers not different in number from those of normal mouse were detected at the dose of 5 mg gentamicin.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.