CHIMERIC PROTEINS BASED ON HUMAN UTROPHIN AND DYSTROPHIN AND USE THEREOF FOR TREATING DUCHENNE MUSCULAR DYSTROPHY

摘要

FIELD: biotechnology; medicine.;SUBSTANCE: group of inventions relates to biotechnology and medicine, particularly to chimeric proteins containing human dystrophin and utrophin sequences. Disclosed is a chimeric protein for treating Duchenne muscular dystrophy, comprising an N-terminal domain of human utrophin, in which a peptide encoded by a first exon of utrophin is optionally replaced with a peptide, encoded by a first exon of human dystrophin, a first unstructured domain of human dystrophin (hinge 1), spectrin repeats 1, 2, 3 of human utrophin, a second unstructured domain of human utrophin (hinge 2), spectrin repeat 22 of human utrophin, fourth unstructured domain of human utrophin (hinge 4), cysteine-rich (CR) domain of human utrophin, oligopeptide QAM encoded by exon 74 of human utrophin. Also disclosed are nucleic acids coding said proteins and viral expression vectors for introduction into a mammal body, containing said nucleic acids.;EFFECT: inventions can be used for gene replacement therapy of Duchenne muscular dystrophy when delivered into muscle cells of patients using known expression vectors and further expression therein of a chimeric protein capable of functionally replacing absent dystrophin.;8 cl, 10 dwg, 3 ex