公开(公告)号：US20240238444A1

公开(公告)日：2024-07-18

申请号：US18310969

申请日：2023-05-02

Applicant: KOREA RESEARCH INSTITUTE OF BIOSCIENCE AND BIOTECHNOLOGY

Inventor： Sun Uk KIM ,  Young Ho PARK ,  Seung Hwan LEE ,  Jong Hee LEE ,  Jae Jin CHA ,  Han Seop KIM ,  Un Bin CHAE

IPC: A61K48/00 ,  A61K31/7105 ,  A61K38/46 ,  A61P39/00 ,  C12N9/22 ,  C12N15/11

CPC classification number: A61K48/0033 ,  A61K31/7105 ,  A61K38/465 ,  A61P39/00 ,  C12N9/22 ,  C12N15/11 ,  C12N2310/20

Abstract: Provided is a composition for the prevention or treatment of Hutchinson-Gilford Progeria syndrome (HGPS) using gene editing, which contains sgRNA that hybridizes to mRNA encoding progerin, which causes HGPS, and a gene encoding Cas13 protein acting on the same. When introduced into the cell of a subject to be treated and only the mRNA encoding progerin is selectively cut. There is no need for co-prescribing with other therapies and fewer side effects occur than traditional farnesyltransferase inhibitors (FTIs). The efficiency is higher than when treated using homologous recombination (HR) at the DNA level, treatment using composition can be made reversibly, and the composition can be applied specifically compared to targeted treatment using RNAi (RNA interference), and has fewer side effects. Compared to treatment using CRISPR/Cas9, which directly acts on DNA and produces irreversible results, treatment using composition is reversible and selectively cut only mRNA encoding progerin, thereby ensuring safety.

公开(公告)号：US20210392863A1

公开(公告)日：2021-12-23

申请号：US17288045

申请日：2019-10-23

Applicant: KOREA RESEARCH INSTITUTE OF BIOSCIENCE AND BIOTECHNOLOGY

Inventor： Sun Uk KIM ,  Young Ho PARK ,  Kyu Tae CHANG ,  Bo Woong SIM ,  Bong Seok SONG ,  Hae Jun YANG ,  Sang Rae LEE ,  Kang Jin JEONG ,  Pil Soo JEONG ,  Yeung Bae JIN ,  Phil Yong KANG ,  Seung Hwan LEE ,  Hwal Yong LEE ,  Kyung Seob LIM ,  Young Hyun KIM ,  Ji Su KIM ,  Han Na KIM ,  Hee Chang SON ,  Seung Bin YOON ,  Jong Hee LEE ,  Seon A CHOI ,  Jae Won HUH

IPC: A01K67/027 ,  C12N15/11 ,  C12N9/22 ,  C12N15/85

Abstract: The present disclosure relates to a dwarfism animal model carrying an IGF-1 gene mutation and a method for generating the same. According to the present disclosure, the problem that an animal dies immediately after birth is overcome, the majority of phenotypes seen in Laron syndrome patients may be observed in the dwarfism animal model, and the dwarfism animal model has decreased expression of personality genes. Thus, the dwarfism animal model may be effectively used as a dwarfism-related disease model.

公开(公告)号：US20240008460A1

公开(公告)日：2024-01-11

申请号：US18034995

申请日：2021-10-15

Applicant: KOREA RESEARCH INSTITUTE OF BIOSCIENCE AND BIOTECHNOLOGY

Inventor： Sun Uk KIM ,  Young Ho PARK ,  Bo Woong SIM ,  Kyu Tae CHANG ,  Seung Hwan LEE ,  Bong Seok SONG ,  Pil Soo JEONG ,  Hae Jun YANG ,  Sang Rae LEE ,  Yeung Bae JIN ,  Kang Jin JEONG

IPC: A01K67/027 ,  C12N9/22 ,  C12N15/11 ,  C12N15/90

CPC classification number: A01K67/0276 ,  C12N9/22 ,  C12N15/11 ,  C12N15/907 ,  A01K2227/108 ,  A01K2217/075 ,  A01K2217/054 ,  A01K2267/0387 ,  C12N2310/20

Abstract: The present disclosure relates to a JAK3 gene-mutated severe combined immunodeficiency animal model and a method of constructing the same. In the JAK3 gene-mutated severe combined immunodeficiency animal model of the present disclosure, the JAK3 gene is specifically deficient, the expression of cytokines is regulated by controlling the number and activity of macrophages, and the thymus, lymphocytes, and Peyer's patches, which are observed in conventional severe combined immunodeficiency animal models, particularly mini-pigs, are completely lacking. In addition, the animal model of the present disclosure can be used as a treatment model for JAK3 SCID patients, as similar phenotypes are observed in patients with human severe combined immunodeficiency caused by a JAK3 gene mutation, and can be used for artificial blood development or xenotransplantation.