Patent ID: 7763656

Claim:
A method for increasing the activity of a protein encoded by an mRNA that contains a premature stop codon, comprising contacting in vitro a cell containing the mRNA with an effective amount of a compound of the formula: or a pharmaceutically acceptable salt thereof wherein: X is oxygen, sulfur, CO, SO, or S(O ) 2 ; Y is oxygen or sulfur; Z is substituted or unsubstituted aryl, substituted or unsubstituted heteroaryl, or substituted or unsubstituted cycloalkyl; n is an integer from 0 to 4; R 1 is hydrogen, substituted or unsubstituted alkyl, substituted or unsubstituted alkenyl, substituted or unsubstituted alkynyl, substituted or unsubstituted cycloalkyl, substituted or unsubstituted heterocycloalkyl, substituted or unsubstituted aryl, substituted or unsubstituted heteroaryl, SO 2 R 7 , CF 3 , CN, COOH, COR 7 , or COOR 7 ; R 0 is hydrogen, or taken together with R 1 and the atoms to which they are attached form an optionally substituted 5-7 membered heterocyclic, or heteroaryl ring; R 2 , R 3 , R 4 , and R 5 are independently hydrogen, substituted or unsubstituted alkyl, substituted or unsubstituted alkenyl, substituted or unsubstituted alkynyl, substituted or unsubstituted cycloalkyl, substituted or unsubstituted heterocycloalkyl, substituted or unsubstituted aryl, substituted or unsubstituted heteroaryl, aryloxy, heteroaryloxy, halogen, CF 3 , OCF 3 , OCHF 2 , CN, COOH, COOR 7 , SO 2 R 7 , NO 2 , NH 2 , or N(R 7 ) 2 ; R 6 is hydrogen, substituted or unsubstituted alkyl, substituted or unsubstituted alkenyl, substituted or unsubstituted alkynyl, substituted or unsubstituted cycloalkyl, substituted or unsubstituted heterocycloalkyl, substituted or unsubstituted aryl, substituted or unsubstituted heteroaryl, or any biohydrolyzable group; each occurrence of R 7 is independently hydrogen, substituted or unsubstituted alkyl, substituted or unsubstituted alkenyl, substituted or unsubstituted alkynyl, substituted or unsubstituted cycloalkyl, substituted or unsubstituted heterocycloalkyl, substituted or unsubstituted aryl, substituted or unsubstituted heteroaryl, alkoxy, aryloxy, heteroaryloxy, halogen, or CF 3 ; and wherein the activity of the protein is increased in the presence of the compound relative to the absence of the compound.