Aminoglycoside derivatives and uses thereof in treating genetic disorders

A compound represented by general formula I:

• or a pharmaceutically acceptable salt thereof,
• wherein:
• the dashed lines indicates a stereo-configuration of position 6′ being an R configuration or an S configuration;
• R1 is alkyl, cycloalkyl or aryl;
• R2 is selected from a substituted or unsubstituted alkyl, OR′ and NR′R″, wherein each of R′ and R″ is independently selected from hydrogen, a substituted or unsubstituted alkyl, a substituted or unsubstituted cycloalkyl, a substituted or unsubstituted aryl, a substituted or unsubstituted heteroaryl, a substituted or unsubstituted alkaryl, and an acyl;
• R4 is selected from hydrogen, acyl, an amino-substituted alpha-hydroxy acyl, a substituted or unsubstituted alkyl, a substituted or unsubstituted cycloalkyl, a substituted or unsubstituted aryl, a substituted or unsubstituted alkaryl and a cell-permealizable;
• and
• R3 is hydrogen or a monosaccharide moiety represented by Formula II:
• wherein the curved line denotes a position of attachment;
• and
• R5 and R6 are each independently selected from hydrogen, a substituted or unsubstituted alkyl, a substituted or unsubstituted cycloalkyl, a substituted or unsubstituted aryl, a substituted or unsubstituted alkaryl, a substituted or unsubstituted heteroaryl, acyl, and a cell-permealizable group, or, alternatively, R5 and R6 form together a heterocyclic ring,
• provided that:
• the compound is not gentamycin, geneticin, fortimycin;
• and
• when R2 is hydroxy or NR′R″, and R and R″ are each hydrogen, R4 is not hydrogen, AHB or AHP, and/or at least one of R5 and/or R6, if present, is not hydrogen.