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AMINOGLYCOSIDES AND USES THEREOF IN TREATING GENETIC DISORDERS
专利权人:
Technion Research & Development Foundation Limited
发明人:
Timor BAASOV,Dana ATIA-GLIKIN,Jeyakumar KANDASAMY,Valery BELAKHOV
申请号:
US15453990
公开号:
US20170182078A1
申请日:
2017.03.09
申请国别(地区):
US
年份:
2017
代理人:
摘要:
A new class of pseudo-trisaccharide aminoglycosides having an alkyl group at the 5″ position, exhibiting efficient stop codon mutation readthrough activity, low cytotoxicity and high selectivity towards eukaryotic translation systems are provided. Also provided are pharmaceutical compositions containing the same, and uses thereof in the treatment of genetic disorders, as well as processes of preparing these aminoglycosides. The disclosed aminoglycosides can be represented by the general formula I:or a pharmaceutically acceptable salt thereof, wherein R1 is selected from the group consisting of alkyl, cycloalkyl and aryl; and all other variables and features are as described in the specification.
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