FIELD: biotechnology.
SUBSTANCE: following is described: a group of inventions including a capsid-free viral vector based on closed-ended DNA (ccDNA) for the expression of phenylalanine hydroxylase (PHA) protein, a method of expressing the PHA protein in a cell, including bringing the cell into contact with the above vector, a method of treating a subject suffering from phenylketonuria (PKU), a pharmaceutical composition for gene therapy, a cell for producing RAS protein, a composition for delivering a cccDNA vector, a kit for delivering a cccDNA vector.
EFFECT: invention expands the range of agents for expressing PHA protein.
59 cl, 14 dwg, 15 tbl, 15 ex
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