FIELD: biotechnology.
SUBSTANCE: invention relates to the delivery (transport) of therapeutic enzymes, which can be used in medicine. The invention relates to a compound containing a therapeutic enzyme and a transport element connected to each other directly or via a linker, wherein the transport element is a Fab fragment of an IgG immunoglobulin specific for an epitope in the insulin receptor, to the use of the said compound for the preparation of a pharmaceutical composition for the treatment of diseases and the use of the said compound for the treatment and prevention of diseases, in particular lysosomal storage diseases, including for the treatment and prevention of deficiency of the corresponding enzyme characteristic of the corresponding lysosomal storage disease, such as mucopolysaccharidosis, in particular mucopolysaccharidosis of types I and II.
EFFECT: obtaining a compound containing a therapeutic enzyme and a transport element connected to each other directly or by using a linker.
15 cl, 4 ex, 5 tbl, 3 dwg
| Title | Year | Author | Number |
|---|---|---|---|
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| METHOD FOR PRODUCING A POLYPEPTIDE HETEROMULTIMER | 2014 |
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| METHOD OF PRODUCING A POLYPEPTIDE HETEROMULTITERS | 2014 |
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| TUMOR-TARGETED AGONISTIC CD28-ANTIGEN-BINDING MOLECULES | 2019 |
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