Method for enhancing mutant enzyme activities in lysosomal storage disorders

A method of enhancing in a mammalian cell the activity of an enzyme associated with a lysosomal storage disorder, wherein a level of said enzyme is deficient in said lysosomal storage disorder, said method comprising administering a reversible competitive inhibitor of said enzyme in an amount effective to enhance the activity of the enzyme, and wherein said reversible competitive inhibitor is a specific competitive inhibitor for said deficient enzyme associated with said lvsosomal storage disorder, wherein said competitive inhibitor is represented by the formula:

• wherein R1 and R1′ ndependently represent H, OH, a 1-12 carbon alkyl, hydroxyalkyl or an alkoxyl group;
• R2 and R2′ independently represent H, OH, or N-acetamido group, or a 1-12 carbon alkyl group;
• R4 and R4′ independently represent H, OH;
• R6 and R6′ independently represent H, CH2OH, CH3, or COOH;
• R7 represent H, or OH;
• R0 represents H, methyl, or a straight chain or branched saturated or unsaturated carbon chain containing 9-12 carbon atoms, optionally substituted with a phenyl, hydroxyl or cyclohexyl group.

A method of treating a lysosomal storage disorder comprising administering an effective amount of a compound of the formula

• wherein R1 and R1′ independently represent H, OH, a 1-12 carbon alkyl, hydroxyalkyl or alkoxy group;
• R2 and R2′ independently represent H, OH, or N-acetamido group, or a 1-12 carbon alkyl group;
• R4 and R4′ independently represent H, OH;
• R6 and R6′ independently represent H, CH2OH, CH3, or COOH;
• R7 represents H, or OH;
• R0 represents H, methyl, or a straight chain or branched saturated or unsaturated carbon chain containing 9-12 carbon atoms, optionally substituted with a phenyl, hydroxyl or cyclohexyl group.

A method of enhancing the activity of an enzyme associated with a lysosomal storage disorder in mammalian cells, wherein a level of said enzyme is deficient in said lysosomal storage disorder, comprising administering an effective amount of a compound of the formula

• wherein R1 and R1′ independently represent H, OH, a 1-12 carbon alkyl, hydroxyalkyl or alkoxy group (preferably 1-3);
• R2 and R2′ independently represent H, OH, or N-acetamido group, or
• a 1-12 carbon alkyl group;
• R4 and R4′ independently represent H, OH;
• R6 and R6′ independently represent H, CH2OH, CH3, or COOH;
• R7 represents H, or OH;
• R0 represent H, methyl, or a straight chain or branched saturated or unsaturated carbon chain containing 9-12 carbon atoms, optionally substituted with a phenyl, hydroxyl or cyclohexyl group.