公开(公告)号：US20220204955A1

公开(公告)日：2022-06-30

申请号：US17606502

申请日：2019-04-26

Applicant: TOOLGEN INCORPORATED

Inventor： Joon Sun LEE ,  Min Hee JUNG ,  Yu Ri CHOI ,  Jeong Joon LEE

IPC: C12N9/22 ,  C12N15/11 ,  C12N15/88 ,  C12N15/85

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.

公开(公告)号：US20220192163A1

公开(公告)日：2022-06-23

申请号：US17440350

申请日：2020-03-24

Applicant: TOOLGEN INCORPORATED

Inventor： Jae Young LEE ,  Hee Sook BAE ,  Hye Jung SHIN ,  Dong Woo SONG ,  Un Gi KIM ,  Kyu Jun LEE

IPC: A01K67/027 ,  C12N15/10 ,  C12N15/113 ,  C12N15/85 ,  C12N15/90 ,  C12N9/64

Abstract: The present invention relates to a hemophilia B rat and a method of producing the hemophilia B rat. More particularly, the present invention relates to a hemophilia B rat having F9 factor knocked-down or knocked-out and a method of producing the hemophilia B rat.

公开(公告)号：US20210254086A1

公开(公告)日：2021-08-19

申请号：US16973140

申请日：2019-06-10

Applicant: TOOLGEN INCORPORATED

Inventor： Geung Joo LEE ,  Luhua TU ,  Saminathan SUBBURAJ

IPC: C12N15/82 ,  C12N15/113 ,  C12N9/22

Abstract: The present application relates to a method for inducing flavonoid biosynthetic gene-editing in a cell by using a CRISPR/CAS9 system.

公开(公告)号：US20200263204A1

公开(公告)日：2020-08-20

申请号：US16642348

申请日：2018-09-28

Applicant: TOOLGEN INCORPORATED

Inventor： Seokjoong KIM ,  Dong Woo SONG ,  Kyu Jun LEE ,  Jung-Min LEE

IPC: C12N15/90 ,  C12N9/22 ,  C07K14/755 ,  C12N15/86 ,  A61P7/04

Abstract: The present invention relates to a CRISPR/Cas system having an inversion correction potential, which uses at least one guide RNA targeting a sequence region where two different homologs present on genomic introns are conjugated to each other in an inversion manner, and a Cas protein, and a CRISPR/Cas system of FVIII gene inversion correction potential that uses at least one guide RNA targeting an int22-1/3 homolog or int22-1/2 homolog sequence region present on intron 22 of coagulation factor VIII (F8) gene and a Cas protein. A CRISPR/Cas system according to the present invention comprises a system which employs a small-size Cas9 and a guide RNA fitted thereto, thereby enabling all CRISPR/Cas instruments to be easily packaged in one AAV, which is impossible in conventional large-size Cas9. In addition, the CRISPR/Cas system can induce normal gene expression thanks to the inversion gene correction potential thereof and is excellent as a technology capable of effectively overcoming the difficult intracellular delivery of large-size gene mutation through gene editing. Particularly, the system can induce normal FVIII expression by restoring the inversion of FVIII gene and thus is useful for the treatment of hemophilia A.

公开(公告)号：US20190177710A1

公开(公告)日：2019-06-13

申请号：US16310114

申请日：2017-06-14

Applicant: TOOLGEN INCORPORATED

Inventor： Jeong Joon LEE

IPC: C12N9/22 ,  C12Q1/44 ,  C12N15/11

Abstract: The present invention relates to a method for selecting a target specific nuclease having high specificity and high activity and, more particularly, to a method for screening and selecting a target specific nuclease system by using a multiple target system capable of simultaneously identifying off-target activity and on-target activity. Through the present invention, it is possible to select a target specific nuclease which has a decreased off-target effect and an increased on-target effect.

公开(公告)号：US20150344912A1

公开(公告)日：2015-12-03

申请号：US14685510

申请日：2015-04-13

Applicant: TOOLGEN INCORPORATED

Inventor： Jin-Soo Kim ,  Seung Woo Cho ,  Sojung Kim

IPC: C12N15/90

CPC classification number: C12N15/52 ,  C12N9/16 ,  C12N9/22 ,  C12N15/102 ,  C12N15/111 ,  C12N15/63 ,  C12N15/8216 ,  C12N15/85 ,  C12N15/907 ,  C12N2310/10 ,  C12N2310/20 ,  C12N2310/531 ,  C12Y301/21

Abstract: The present invention relates to targeted genome editing in eukaryotic cells or organisms. More particularly, the present invention relates to a composition for cleaving a target DNA in eukaryotic cells or organisms comprising a guide RNA specific for the target DNA and Cas protein-encoding nucleic acid or Cas protein, and use thereof.