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.