ENHANCEMENT OF TISSUE-SPECIFIC GENE DELIVERY BY CAPSID MODIFICATION Russian patent published in 2023 - IPC C07K14/10 C12N15/86 A61K31/7088 A61K38/46 

Abstract RU 2801217 C2

FIELD: biotechnology.

SUBSTANCE: following is provided: a modified VP1 AAVrh74 capsid protein for increased gene delivery comprising either one or more modifications selected from isoleucine to asparagine substitution at amino acid position 502, tryptophan to arginine substitution at position 505 of VP1 AAVrh74, and insertion of a YIG or YIGSR peptide (SEQ ID NO: 2) to the position of amino acid 591 VP1 of AAVrh74 or their equivalents. Also a recombinant viral particle for increased gene delivery containing a modified AAVrh74 VP1 is presented. Besides, a polynucleotide encoding a modified AAVrh74 VP1 and a gene delivery vector containing said polynucleotide are described. A polynucleotide encoding a recombinant viral particle and a gene delivery vector containing the described polynucleotide, as well as a recombinant expression system containing the described polynucleotide, a recombinant expression system containing the vector are described. Also a host cell containing a polynucleotide is provided. A method of modifying a polynucleotide in a cell or in a subject, which includes delivering an effective amount of a recombinant viral particle into the cell is described. A kit for modifying a polynucleotide, including a recombinant viral particle is presented.

EFFECT: invention expands the arsenal of agents for therapeutic use.

19 cl, 5 dwg, 1 tbl, 1 ex

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RU 2 801 217 C2

Authors

Loiler, Scott Allen

Dates

2023-08-03Published

2019-03-14Filed