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...

Ausführliche Beschreibung

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Bibliographische Detailangaben
Hauptverfasser: FIRZON, Vera, BAASOV, Timor, EYLON, Bat-Hen, BELAKHOV, Valery
Format: Patent
Sprache:eng ; fre ; ger
Schlagworte:
CHEMISTRY
DERIVATIVES THEREOF
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
METALLURGY
NUCLEIC ACIDS
NUCLEOSIDES
NUCLEOTIDES
ORGANIC CHEMISTRY
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
SUGARS
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Beschreibung
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.