LIVER-SPECIFIC STRUCTURES, EXPRESSION CASSETTES OF FACTOR VIII AND THEIR APPLICATION METHODS Russian patent published in 2022 - IPC C12N15/113 C12N15/63 C12N15/861 A61K38/37 A61K48/00 A61P7/02 

Abstract RU 2774874 C2

FIELD: gene therapy.

SUBSTANCE: group of inventions relates to gene therapy, in particular to target delivery of transgene-encoding structures to the liver for the expression of therapeutic proteins. Polynucleotide expression structures, and AAV vector containing a polynucleotide expression structure are proposed. A pharmaceutical composition for the treatment of hemophilia in a subject who needs it, and a pharmaceutical composition for the provision of a transgene that encodes a human Factor VIII for a subject who needs it, containing the specified AAV vector are also proposed. A method for the provision of a human Factor VIII for a subject who needs it, and a method for the induction of tolerance in a subject to a human Factor VIII are also proposed.

EFFECT: group of inventions provides for the expression of transgenes at high levels in liver cells, using the specified expression structures.

15 cl, 40 dwg, 9 tbl, 3 ex

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RU 2 774 874 C2

Authors

Rajli, Bridzhit E.

Li, Geri K.

Dates

2022-06-23Published

2016-07-13Filed