HSD17B13 OPTIONS AND THEIR APPLICATIONS Russian patent published in 2021 - IPC C12N15/113 C12N15/85 

Abstract RU 2760851 C2

FIELD: biotechnology.

SUBSTANCE: invention relates to the field of biotechnology, in particular to a method for modifying HSD17B13 gene in a cell, including the introduction into the cell of Cas9 protein or nucleic acid encoding Cas9 protein and the first guide RNA or DNA encoding the first guide RNA, which forms a complex with Cas9 protein and targets the first target sequence for the guide RNA in HSD17B13 gene. A method for changing the expression of HSD17B13 gene in a cell is also disclosed, including the introduction into the cell of Cas9 protein or nucleic acid encoding Cas9 protein, wherein Cas9 protein is non-nuclease-active Cas9 protein fused with a transcription repressor domain or a transcription activator domain, and the first guide RNA or DNA encoding the first guide RNA targeting the first target sequence for the guide RNA in HSD17B13 gene.

EFFECT: invention is effective for the therapy or prevention of liver diseases.

81 cl, 17 dwg, 13 tbl, 4 ex

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RU 2 760 851 C2

Authors

Abul-Husn, Noura, S.

Gottesman, Omri

Li, Alexander

Cheng, Xiping

Xin, Yurong

Pefanis, Evangelos

Hartford, Suzanne

Gromada, Jesper

Dewey, Frederick, E.

Baras, Aris

Shuldiner, Alan

Dates

2021-11-30Published

2018-01-19Filed