METHOD FOR PRODUCING FUNCTIONAL PROTEIN FROM DNA HAVING NONSENSE MUTATIONS, AND TREATING ASSOCIATED DISORDERS Russian patent published in 2012 - IPC A61K31/4245 A61P11/00 A61P21/00 A61P43/00 

FIELD: medicine, pharmaceutics.

SUBSTANCE: group of invention refers to medicine, and concerns treating nonsense mutation(s) associated diseases: Duchene muscular dystrophy and cystic fibrosis. What is presented is the use of 3-[5-(2-fluorophenyl)-[1,2,4]oxadiazole-3-yl]benzoic acid or its pharmaceutically acceptable salts, solvate or hydrate in preparing a drug for treating Duchene muscular dystrophy associated with nonsense mutation in position 1417, 3625 or 492 of dystrophin; and cystic fibrosis associated with nonsense mutation in position 414, 493, 1316, 553, 542, 1162, 122, 1455, 822, 60, 764, 1291, 849, 434, 88, 1158 or 6542 CTFR. What is also presented is the use of 3-[5-(2-fluorophenyl)-[1,2,4]oxadiazole-3-yl]benzoic acid or its pharmaceutically acceptable salts, solvate or hydrate in preparing a drug for producing a functional readthrough protein in a subject having Duchene muscular dystrophy and cystic fibrosis.

EFFECT: use of 3-[5-(2-fluorophenyl)-[1,2,4]oxadiazole-3-yl]benzoic acid provides suppression of premature termination of translation in a subject by mediating nonsense codon reading and producing the functional protein in the amount sufficient for treating the disease.

3 cl, 3 dwg, 17 tbl, 16 ex