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公开(公告)号:US20200172912A1
公开(公告)日:2020-06-04
申请号:US16700942
申请日:2019-12-02
Applicant: TOOLGEN INCORPORATED
Inventor: Eun Ji Kim , Seok Joong Kim
Abstract: The disclosure provided herewith relates to a Campylobacter jejuni CRISPR/CAS system-derived RGEN and a use thereof.
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公开(公告)号:US20190276503A1
公开(公告)日:2019-09-12
申请号:US16349672
申请日:2017-09-28
Applicant: TOOLGEN INCORPORATED
Inventor: Seok Joong KIM , Dong Woo SONG , Young Bin HONG , Byung Ok CHOI , Jae Young LEE , Jung Min LEE
IPC: C07K14/315 , C12N15/86 , A61K35/30
Abstract: The present invention relates to an artificially manipulated SC function-controlling factor for SC function control and/or the treatment or alleviation of a disease due to an SC function disorder, and to a use thereof. More specifically, the present invention relates to a system capable of performing artificial SC function control and/or treating or alleviating a disease due to an SC function disorder, the system comprising: an artificially manipulated SC function-controlling factor for SC function control and/or the treatment or alleviation of a disease due to an SC function disorder; and/or a composition for treating or alleviating a disease due to an SC function disorder. In a specific aspect, the present invention relates to an SC function-controlling system by an SC function-controlling factor, such as artificially manipulated PMP22, and/or an expression product thereof.
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公开(公告)号:US20190185860A1
公开(公告)日:2019-06-20
申请号:US16324955
申请日:2017-08-14
Applicant: TOOLGEN INCORPORATED
Inventor: Seok Joong KIM , Yoon-Young KIM , Ho-Sung YU , In-Young JUNG , Jung Min LEE
IPC: C12N15/113 , C12N15/85 , C12N5/0784 , C12N5/0783 , C12N9/22 , C12N15/52
CPC classification number: C12N15/1132 , A61K35/14 , C12N5/0637 , C12N5/0638 , C12N5/064 , C12N5/0646 , C12N9/22 , C12N15/52 , C12N15/63 , C12N15/85 , Y02A50/47
Abstract: The present invention relates to an artificially manipulated immune system having an improved immune effect. More particularly, the present invention relates to an immune system having functions artificially altered which comprises artificially manipulated immunoregulatory elements and cells containing the same. Contemplated according to a particular embodiment is an immune system comprising artificially manipulated immunoregulatory genes such as PD-1, CTLA-4, A20, DGKα, DGKζ, FAS, EGR2, PPP2R2D, PSGL-1, KDM6A, and TET2, and/or expression products thereof.
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公开(公告)号:US20250146002A1
公开(公告)日:2025-05-08
申请号:US19013018
申请日:2025-01-08
Applicant: TOOLGEN INCORPORATED
Inventor: Jin-Soo Kim , Seung Woo Cho , Sojung Kim
IPC: C12N15/52 , C12N9/16 , C12N9/22 , C12N15/10 , C12N15/11 , C12N15/63 , C12N15/82 , C12N15/85 , C12N15/90
Abstract: The present disclosure relates to targeted genome editing in eukaryotic cells or organisms. More particularly, the present disclosure provides for compositions and methods that may induce modifications in target endogenous nucleic acid sequences in nucleuses of eukaryotic cells. For example, disclosed here in is a method of introducing a site-specific, double-stranded break at a target nucleic acid sequence in a eukaryotic cell, the method comprising introducing into the eukaryotic cell a Type II Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/Cas system, wherein the CRISPR/Cas system comprises: a) a nucleic acid encoding a Cas9 polypeptide, wherein the Cas9 polypeptide comprises a nuclear localization signal, and b) a chimeric guide RNA comprising a CRISPR RNA (crRNA) portion fused to a trans activating crRNA (tracrRNA) portion, wherein the target nucleic acid sequence comprises a first strand having a region complementary to the crRNA portion of the chimeric guide RNA and a second strand having a trinucleotide protospacer adjacent motif (PAM), and whereby the site-specific, double-stranded break at the target nucleic acid sequence is introduced in the eukaryotic cell.
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公开(公告)号:US20250115906A1
公开(公告)日:2025-04-10
申请号:US18727149
申请日:2023-01-12
Applicant: TOOLGEN INCORPORATED
Inventor: Dong Woo SONG , Hyerim LEE , Hye-Kyung OH , Beom Seok CHOI , Kyu Jun LEE , Nan Yeong GO , Jae Young LEE
IPC: C12N15/113 , C12N9/22 , C12N15/11 , C12N15/86
Abstract: The present invention relates to a Schwann cell-specific promoter and use thereof. More particularly, the present invention relates to an artificially engineered minimal promoter that specifically operates in Schwann cells, wherein the artificially engineered minimal promoter is derived from an MPZ (Myelin Protein Zero or PO) promoter. In addition, the present invention relates to a vector using the Schwann cell-specific promoter and a method for treating Schwann cell-related diseases using same.
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公开(公告)号:US20240216539A1
公开(公告)日:2024-07-04
申请号:US18590251
申请日:2024-02-28
Inventor: Jae Young Lee , Sang Woo Ham , Hee Sook Bae , Gyu Bon Cho , Jae Hyung Jang , Yun Ha Kim , Dong Soo Lee
CPC classification number: A61K48/005 , A61K47/6901 , C12N15/86 , C12N2750/14143
Abstract: The present application relates to a vector for delivering an active agent to the nervous system. In one embodiment of the present application, a vector for delivering an active agent in which one or more TTR ligands are conjugated to the surface of an adeno-associated virus is provided.
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公开(公告)号:US20230416315A1
公开(公告)日:2023-12-28
申请号:US18031202
申请日:2021-11-03
Applicant: TOOLGEN INCORPORATED
Inventor: Sang Woo HAM , Jae Young LEE
CPC classification number: C07K14/47 , A61K38/1709 , A61P25/28 , C12N15/86 , C07K2319/30 , C07K2319/02 , A61K48/00
Abstract: The present invention relates to prion-Fc region fusion proteins and vectors capable of expressing the prion-Fc region fusion proteins. More particularly, the prion-Fc region fusion protein is a fusion protein comprising the Fc region of a human prion protein and an immunoglobulin. The prion-Fc region fusion protein can function to act on and remove the neurotoxic protein aggregates described above. In addition, the present invention relates to a use the prion-Fc region fusion proteins, or vectors capable of expressing the same, and a method for treating diseases associated with neurotoxic protein aggregates.
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公开(公告)号:US11845951B2
公开(公告)日:2023-12-19
申请号:US16650993
申请日:2018-09-28
Applicant: TOOLGEN INCORPORATED , SEOUL NATIONAL UNIVERSITY R&DB FOUNDATION , SEOUL NATIONAL UNIVERSITY HOSPITAL
Inventor: Dong Woo Song , Jung Min Lee , Un Gi Kim , Jeong Hun Kim , Dong Hyun Jo
IPC: C12N15/86 , C12N15/113 , C12N9/22 , A61P27/02 , A61K48/00
CPC classification number: C12N15/86 , A61P27/02 , C12N9/22 , C12N15/113 , A61K48/00 , C12N2310/20 , C12N2800/80
Abstract: The present invention relates to a composition for gene manipulation for treating or improving a retinal dysfunction disease or a method using the same. More particularly, the present invention relates to a composition for gene manipulation including a guide nucleic acid capable of targeting a retinal function-forming gene and a method of treating or improving a disease caused by retinal dysfunction by artificially manipulating and/or correcting a retinal function-forming gene using the same.
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公开(公告)号:US20230130858A1
公开(公告)日:2023-04-27
申请号:US17880160
申请日:2022-08-03
Applicant: TOOLGEN INCORPORATED
Inventor: Young-Hoon KIM , Min Hee JUNG , Joonsun LEE , Eunji SHIN , Kang In LEE , Seokjoong KIM , Jeongjoon LEE
Abstract: The present invention relates to an artificially engineered CRISPR/Cas9 system. More particularly, the present invention relates to an artificially engineered CRISPR enzyme having enhanced target specificity and a use of an artificially engineered CRISPR/Cas9 system including the same enzyme in genome and/or epigenome manipulation or modification, genome targeting, genome editing, and in vitro diagnosis, etc.
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公开(公告)号:US11441135B2
公开(公告)日:2022-09-13
申请号:US16628199
申请日:2018-07-06
Applicant: TOOLGEN INCORPORATED
Inventor: Young-Hoon Kim , Min Hee Jung , Joonsun Lee , Eunji Shin , Kang In Lee , Seokjoong Kim , Jeongjoon Lee
Abstract: The present invention relates to an artificially engineered CRISPR/Cas9 system. More particularly, the present invention relates to an artificially engineered CRISPR enzyme having enhanced target specificity and a use of an artificially engineered CRISPR/Cas9 system including the same enzyme in genome and/or epigenome manipulation or modification, genome targeting, genome editing, and in vitro diagnosis, etc.
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