GENE THERAPY MONITORING Russian patent published in 2024 - IPC C12N15/00 A61K48/00 G01N33/50 G01N33/68 

Abstract RU 2825840 C2

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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RU 2 825 840 C2

Authors

Chau, B. Nelson

Liao, Jing

Gordo, Susana

Dates

2024-09-02Published

2020-04-14Filed