A Structure-Activity Relationship(SAR) study was carried out to identify novel therapeutic compounds that allowreadthrough of premature termination codons (PTCs) for treating geneticdiseases resulting from nonsense mutations, such as Duchenne's musculardystrophy (DMD) and ataxia-telangiactasia (A-T).  It has been demonstrated that certain compoundscan induce readthrough of PTCs, resulting in translation of full-length functionalprotein.  Our research group has recentlydeveloped non-aminoglycoside readthrough compounds (RTCs) showing excellentreadthrough activity. Current efforts are focused on compound optimization bypreparing and identifying molecules with improved activity and pharmacokineticproperties. The successful development of novel RTCs is anticipated to demonstratechemical-induced readthrough of PTCs and therefore, potentially resulting in anefficient strategy for treating A-T and DMD genetic diseases.

Synthesis and evaluation of non-aminoglycosides
as potential readthrough compounds

241st National Meeting (2011)

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Synthesis and evaluation of non-aminoglycosides as potential readthrough compounds