Aminoglycoside derivatives and uses thereof in treating genetic disorders

Aminoglycoside derivatives and uses thereof in treating genetic disorders

Novel aminoglycosides, represented by Formulae (e.g., a compound of Formula A, B, I, I*, III or III*, including compounds represented by Formula Ia, I**, I*a, I*b, IIIa, III**, III*a and, III*b), as defined in the instant specification, designed to exhibit stop codon mutation readthrough activity, a...

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Bibliographische Detailangaben
Hauptverfasser: Bat-Hen EYLON, VALERY BELAKHOV, TIMOR BAASOV, Vera FIRZON
Format: Patent
Sprache:eng ; heb
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Zusammenfassung:Novel aminoglycosides, represented by Formulae (e.g., a compound of Formula A, B, I, I*, III or III*, including compounds represented by Formula Ia, I**, I*a, I*b, IIIa, III**, III*a and, III*b), as defined in the instant specification, designed to exhibit stop codon mutation readthrough activity, are provided. Also provided are pharmaceutical compositions containing the same, and uses thereof in the treatment of genetic diseases and disorders, such as diseases and disorders associated with stop codon mutations.