FIELD: biotechnology.
SUBSTANCE: group of inventions including a recombinant adeno-associated virus (rAAV) for delivering a transgene to liver cells (versions), a composition, a method for delivering a transgene, a method for re-introducing a transgene into a cell for expressing a heterologous gene in a cell, using rAAV for obtaining a drug (variants), a method for treating a subject with a disorder or disease that can be treated by liver expression of a transgene, a method for reintroducing a transgene to express a heterologous gene in a cell to treat a subject with a disorder or disease that can be treated by liver expression of the transgene, a method for treating a subject suffering from the disorder or a disease associated with abnormal activity of an endogenous protein, a vector for producing rAAV (variants), a cell for expressing a heterologous gene, a method for producing rAAV.
EFFECT: invention expands the arsenal of means for delivering the transgene to the liver.
47 cl, 14 dwg, 5 tbl, 2 ex
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