公开(公告)号：US20230212615A1

公开(公告)日：2023-07-06

申请号：US18188228

申请日：2023-03-22

申请人： GUANGZHOU REFORGENE MEDICINE CO., LTD.

发明人： Junbin Liang ,  Hui Xu ,  Jiayu Ou ,  Bo Gu

IPC分类号： C12N15/90 ,  C12N15/11 ,  C12N9/22 ,  C12N15/88

CPC分类号： C12N15/907 ,  C12N15/11 ,  C12N9/22 ,  C12N15/88 ,  C12N2310/20 ,  C12N2800/80

摘要： Provided are gRNA that can direct a Cas enzyme to target a CTGF gene and the use thereof, which belongs to the technical field of gene editing. The gRNA may direct the Cas enzyme to perform targeted cleavage on an SMAD binding site region of a CTGF gene promoter, or the gRNA may direct the Cas enzyme to perform targeted cleavage on a CTGF gene exon 2 region. The gRNA can reduce the overexpression of the human CTGF gene via a CRISPR-Cas gene editing system. The above-mentioned gRNA is used for preparing a drug for use against fibrotic diseases.

公开(公告)号：US20240279630A1

公开(公告)日：2024-08-22

申请号：US18652819

申请日：2024-05-02

申请人： GUANGZHOU REFORGENE MEDICINE CO., LTD. ,  ZHEJIANG SYNSORBIO TECHNOLOGY CO., LTD

发明人： Junbin Liang ,  Xingxiang Liang ,  Yang Sun ,  Hui Xu ,  Zhiqin Peng ,  Kaiwei Si ,  Desheng Huangfu

IPC分类号： C12N9/22 ,  C12N15/11 ,  C12N15/90 ,  C12Q1/6816

CPC分类号： C12N9/22 ,  C12N15/11 ,  C12N15/907 ,  C12Q1/6816 ,  C12N2310/20 ,  C12N2320/10

摘要： The present disclosure relates to an isolated Cas13 protein and use thereof. The amino acid sequence of the isolated Cas13 protein comprises a sequence having ≥50% sequence identity with the sequence as shown in any one of SEQ ID NO: 1-SEQ ID NO: 7, and SEQ ID NO: 60. The Cas13 protein is a Cas13 enzyme with an endonuclease activity, which can be used in a CRISPR/Cas system to achieve targeting and modification of a target nucleic acid, enriching the enzymes and systems available in a CRISPR-C as editing system.

公开(公告)号：US20240352460A1

公开(公告)日：2024-10-24

申请号：US18764207

申请日：2024-07-04

申请人： GUANGZHOU REFORGENE MEDICINE CO., LTD.

发明人： Junbin Liang ,  Jiayu Ou ,  Hui Xu ,  Simiao Lin

IPC分类号： C12N15/113

CPC分类号： C12N15/113 ,  C12N2310/14 ,  C12N2310/333 ,  C12N2310/3341 ,  C12N2310/335 ,  C12N2310/531 ,  C12N2320/33

摘要： Provided is snRNA which targets USH2A pre-mRNA. The recognition domain of the snRNA is the reverse complement of a USH2A pre-mRNA sequence. The snRNA binds to the USH2A pre-mRNA to splice and jump exon 13. The snRNA promotes a higher exon 13 skipping efficiency than AON.