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2 816 897

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(51)

IPC

C12N15/85(2006-01-01)

(21) (22)

Application

2022116800, 2022-06-22

(24)

Start date

2022-06-22

(22)

Actual filing date

2022-06-22

(45)

Published

2024-04-08

(72)

Inventor

Shitik Ekaterina MaksimovnaCherepanova Alena IgorevnaIgnatova Iuliia PetrovnaIudkin Dmitrii Vladimirovich

(73)

Holder

Ooo Transgen

GENETIC CONSTRUCT CONTAINING CHIMERIC GUIDE RNA SEQUENCES FOR DELETION OF HUMAN SMN1 GENE IN HUMAN CELL CULTURES Russian patent published in 2024 - IPC C12N15/85

Abstract RU 2816897 C2

FIELD: molecular biotechnology; medicine.

SUBSTANCE: invention concerns a genetic construct carrying guide RNA genes complementary to the flanking regions of exon 7 of the human SMN1 gene. The presence of U6 promoters ensures expression in human cells, and the presence of a constitutive part (scaffold) ensures recognition of guide RNAs synthesized in the cell by nucleases of the CRISPR system that recognize NGG PAM.

EFFECT: invention makes it possible to create cell lines that model human spinal muscular atrophy, which can be used to study therapeutic agents for this disease.

1 cl, 1 dwg

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RU 2 816 897 C2

Authors

Shitik Ekaterina Maksimovna

Cherepanova Alena Igorevna

Ignatova Iuliia Petrovna

Iudkin Dmitrii Vladimirovich

Dates

2024-04-08—Published

2022-06-22—Filed