Treatment of hereditary diseases with gentamicin

Details Treatment of hereditary diseases with gentamicin Treatment of hereditary diseases with gentamicin

A - Human Necessities

61

K

A61K 31/7036 (2006.01) A61P 21/00 (2006.01)

Patent

CA 2292874

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.

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