METHOD OF PRODUCING RECOMBINANT ADENO-ASSOCIATED VIRUSES FOR USE IN GENE THERAPY Russian patent published in 2020 - IPC C12N15/861 

Abstract RU 2716999 C1

FIELD: biotechnology.

SUBSTANCE: invention relates to the biotechnology. In order to express the transgene exclusively in muscle cells to create an adeno-associated virus using an expression vector containing a muscle-specific promoter C5-12, repeating DNA sequences, complementary to the mir-142 and mir-499 microRNA arms sequences, and a packaging plasmid which contains the C5-tataless promoter and human subtype 2 VP3 virus protein, which contains the additional amino acid sequence TGASSLNIAGLS, for the VP3 virus expression.

EFFECT: invention provides preparing viruses for highly targeted and safe expression of target proteins in muscle tissue.

1 cl, 3 dwg, 3 ex

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RU 2 716 999 C1

Authors

Al-Shekhadat Ruslan Ismailovich

Klimov Nikolaj Anatolevich

Kondrashkina Aleksandra Mikhajlovna

Malakhov Ipatij Sergeevich

Kadyrova Renata Anvarovna

Yukhneva Mariya Aleksandrovna

Galochkina Anastasiya Valerevna

Dates

2020-03-17Published

2019-03-13Filed