| Budget Amount *help |
¥4,030,000 (Direct Cost: ¥3,100,000、Indirect Cost: ¥930,000)
Fiscal Year 2014: ¥1,040,000 (Direct Cost: ¥800,000、Indirect Cost: ¥240,000)
Fiscal Year 2013: ¥2,990,000 (Direct Cost: ¥2,300,000、Indirect Cost: ¥690,000)
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| Outline of Final Research Achievements |
A nonsense mutation is a genetic mutation, which replaces codon encoding an amino acid into premature termination codon (PTC). The presence of PTC results in the expression of truncated proteins and hence causes a variety of diseases such as Duchenne muscular dystrophy. Aminoglycoside-induced readthrough of PTC has potential for the treatment of these diseases. Aminoglycosides induce PTC readthorough by decreasing the accuracy of translation by binding to the decoding center of ribosome. A limitation of aminoglycoside as pharmacogenetic agents is risk of significant toxicity. The effect of aminoglycoside is not specific to PTC, causing incorporation of an incorrect amino acid at a sense codon. We examined to design a new molecule that induces gene- and sequence-specific readthrough of PTC by conjugating aminoglycoside with oligonucleotide.
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