FIELD: chemistry; medicine.
SUBSTANCE: invention relates to biochemistry and medicine, in particular to compositions and methods for delivering a therapeutic protein to the central nervous system (CNS) for treating diseases and disorders in which the function of the central nervous system is disturbed, such as the treatment of lysosomal storage diseases.
EFFECT: therapeutic proteins are provided, which are delivered by means of a therapeutically effective amount of a composition comprising a nucleotide coding a therapeutic protein, conjugated with a cell surface receptor binding protein which crosses the blood-brain barrier (BBB).
52 cl, 27 dwg, 9 tbl, 10 ex
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