FIELD: biotechnology.
SUBSTANCE: described is a group of inventions comprising a construct for use in measuring expression of a therapeutic payload and a method for monitoring gene therapy. Construct comprises 5' to 3' polynucleotide sequence coding (a) 5'-homologous arm, (b) coding sequence 2A, coding peptide 2A, where coding sequence 2A contains a nucleotide sequence coding the amino acid sequence SEQ ID NO: 1, (c) a therapeutic payload and (d) 3'-homologous arm, wherein said construct is integrated into an endogenous albumin target site so that the albumin locus is transcribed to form albumin-2A and a therapeutic payload in the form of separate proteins.
EFFECT: invention extends the range of agents for modifying the expression of the therapeutic payload.
20 cl, 17 dwg, 11 ex
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Authors
Dates
2024-09-02—Published
2020-04-14—Filed