FIELD: biotechnology.
SUBSTANCE: composition for use in the treatment of lysosomal storage diseases, containing a polynucleotide encoding a multidomain therapeutic protein containing a delivery domain and an enzymatic domain, and a pharmaceutically acceptable carrier. In one embodiment, the delivery domain binds to an internalization effector, the internalization effector being CD63.
EFFECT: expanding the scope of drugs for the treatment of lysosomal storage diseases.
15 cl, 17 dwg, 18 tbl, 9 ex
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