Title:
COMPOSITION FOR PREVENTING OR TREATING PROGERIA AND NATURAL AGING THROUGH GENE EDITING
Document Type and Number:
WIPO Patent Application WO/2022/098014
Kind Code:
A1
Abstract:
The present invention relates to a composition for preventing or treating progeria through gene editing, the composition containing sgRNA to be hybridized with mRNA, which encodes progeria-causing progerin, and a gene encoding a Cas13 protein acting thereon, and thus only mRNA encoding progerin introduced into cells to be treated are selectively cleaved. The present invention does not require a combined prescription with other therapeutic agents, unlike a conventional method using a farnesyltransferase inhibitor (FTI), and has few side effects. In addition, the present invention has an efficacy higher than that of therapy using homologous recombination (HR) in at the DNA level, can be reversed, can be more specifically applied than targeted therapy using RNA interference (RNAi), and has few side effects. Furthermore, compared with therapy using CRISPR/Cas9, which exhibits irreversible results by directly acting on DNA, the present invention is reversible, and only mRNA encoding progerin can be selectively cleaved to ensure safety.
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Inventors:
KIM SUN UK (KR)
PARK YOUNG HO (KR)
LEE SEUNG HWAN (KR)
LEE JONG HEE (KR)
CHA JAE JIN (KR)
KIM HAN SEOP (KR)
CHAE UN BIN (KR)
PARK YOUNG HO (KR)
LEE SEUNG HWAN (KR)
LEE JONG HEE (KR)
CHA JAE JIN (KR)
KIM HAN SEOP (KR)
CHAE UN BIN (KR)
Application Number:
PCT/KR2021/015531
Publication Date:
May 12, 2022
Filing Date:
November 01, 2021
Export Citation:
Assignee:
KOREA RES INST BIOSCIENCE & BIOTECHNOLOGY (KR)
International Classes:
C12N15/86; A61K31/7088; A61K48/00; A61P39/00; A61P43/00; C12N9/22; C12N15/113
Domestic Patent References:
| WO2019191341A1 | 2019-10-03 |
Foreign References:
| KR20120106286A | 2012-09-26 | |||
| KR20190025821A | 2019-03-12 |
Other References:
BEYRET ERGIN; LIAO HSIN-KAI; YAMAMOTO MAKO; HERNANDEZ-BENITEZ REYNA; FU YUNPENG; ERIKSON GALINA; REDDY PRADEEP; IZPISUA BELMONTE J: "Single-dose CRISPR–Cas9 therapy extends lifespan of mice with Hutchinson–Gilford progeria syndrome", NATURE MEDICINE, NATURE PUBLISHING GROUP US, NEW YORK, vol. 25, no. 3, 18 February 2019 (2019-02-18), New York, pages 419 - 422, XP036722143, ISSN: 1078-8956, DOI: 10.1038/s41591-019-0343-4
SANTIAGO-FERNáNDEZ OLAYA; OSORIO FERNANDO G.; QUESADA VíCTOR; RODRíGUEZ FRANCISCO; BASSO SAMMY; MAESO DANIEL; ROLAS: "Development of a CRISPR/Cas9-based therapy for Hutchinson–Gilford progeria syndrome", NATURE MEDICINE, NATURE PUBLISHING GROUP US, NEW YORK, vol. 25, no. 3, 18 February 2019 (2019-02-18), New York, pages 423 - 426, XP036722141, ISSN: 1078-8956, DOI: 10.1038/s41591-018-0338-6
DATABASE GenBank NLM; . . : "Homo sapiens progerin mRNA, complete cds", XP055928794
SANTIAGO-FERNáNDEZ OLAYA; OSORIO FERNANDO G.; QUESADA VíCTOR; RODRíGUEZ FRANCISCO; BASSO SAMMY; MAESO DANIEL; ROLAS: "Development of a CRISPR/Cas9-based therapy for Hutchinson–Gilford progeria syndrome", NATURE MEDICINE, NATURE PUBLISHING GROUP US, NEW YORK, vol. 25, no. 3, 18 February 2019 (2019-02-18), New York, pages 423 - 426, XP036722141, ISSN: 1078-8956, DOI: 10.1038/s41591-018-0338-6
DATABASE GenBank NLM; . . : "Homo sapiens progerin mRNA, complete cds", XP055928794
Attorney, Agent or Firm:
PCR INTELLECTUAL PROPERTY LAW FIRM (KR)
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