FIELD: medicine.
SUBSTANCE: group of inventions refers to medicine and provides gene therapy compositions for use in treating a metabolic disorder. Adeno-associated virus 1 (AAV1) vector contains a viral expression construct containing a ubiquitous promoter operably linked to a nucleotide sequence which codes fibroblast growth factor 21 (FGF21) expressed in skeletal muscle. Nucleic acid molecule is codon-optimized for expression in a mammal by a nucleotide sequence which codes FGF21. Metabolic disorder is diabetes mellitus, obesity, non-alcoholic steatohepatitis (NASH), liver inflammation or fibrosis, a metabolic disorder associated with aging, cancer. Group of inventions enables prolonged secretion of stable native FGF21 into the bloodstream.
EFFECT: efficiency is maintained even with single introduction of gene transfer vectors.
16 cl, 77 dwg, 1 tbl, 24 ex
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