FIELD: biotechnology.
SUBSTANCE: disclosed are: a biocompatible implantable element for delivering a polypeptide to a subject, which comprises an engineered active cell or a plurality of engineered active cells, which is an engineered retinal pigment epithelium (RPE) cell or an engineered cell derived from an RPE cell, wherein the constructed active cell comprises an exogenous nucleic acid which encodes a polypeptide and comprises certain nucleotide sequences, and a biocompatible implantable element for delivering a polypeptide to a subject is also disclosed, which contains engineered cage ARPE-19 or multiple engineered cells ARPE-19, where the implanted element comprises an enclosing component surrounding the engineered ARPE-19 cell or a plurality of engineered ARPE-19 cells, where the enclosing component contains an alginate and a method for producing a biocompatible implanted element. Also disclosed are a pharmaceutical composition for delivering a polypeptide to a patient, comprising a set of biocompatible implanted elements, and methods of treating haemophilia A and B in a human subject, comprising administering or providing the subject with a biocompatible implanted element.
EFFECT: invention can be used to treat haemophilia A and B in a subject in need thereof.
40 cl, 14 dwg, 3 tbl, 14 ex
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Authors
Dates
2024-09-05—Published
2018-09-27—Filed