Title:
GENE EDITING SYSTEM FOR TREATING DUCHENNE MUSCULAR DYSTROPHY, AND METHOD OF TREATING DISEASE USING SAME
Document Type and Number:
WIPO Patent Application WO/2023/172116
Kind Code:
A1
Abstract:
The present invention relates to a gene editing system for treating Duchenne muscular dystrophy, and a method for treating the disease using same. An aspect of the present invention has the effects of making it possible to package the gene editing system in a single vector by editing the dystrophin gene using a CRISPR/Cas12f1 or TaRGET system, as well as making it possible to produce the dystrophin protein having a normal function by preventing the production of a stop codon of exon 51 through the skipping of exon 51, and thus can be useful for treating Duchenne muscular dystrophy.
Inventors:
KIM YONG SAM (KR)
KIM DO YON (KR)
LEE JEONG MI (KR)
CHIN HYUN JUNG (KR)
KIM DO YON (KR)
LEE JEONG MI (KR)
CHIN HYUN JUNG (KR)
Application Number:
PCT/KR2023/003337
Publication Date:
September 14, 2023
Filing Date:
March 10, 2023
Export Citation:
Assignee:
GENKORE INC (KR)
International Classes:
C12N9/12; A61K48/00; A61P21/04; C12N9/22; C12N15/10; C12N15/113; C12N15/86
Foreign References:
| KR20200116933A | 2020-10-13 | |||
| US20170254799A1 | 2017-09-07 | |||
| KR20210053228A | 2021-05-11 | |||
| KR20210016170A | 2021-02-15 |
Other References:
KIM DO YON; LEE JEONG MI; MOON SU BIN; CHIN HYUN JUNG; PARK SEYEON; LIM YOUJUNG; KIM DAESIK; KOO TAEYOUNG; KO JEONG-HEON; KIM YONG: "Efficient CRISPR editing with a hypercompact Cas12f1 and engineered guide RNAs delivered by adeno-associated virus", NATURE BIOTECHNOLOGY, NATURE PUBLISHING GROUP US, NEW YORK, vol. 40, no. 1, 2 September 2021 (2021-09-02), New York, pages 94 - 102, XP037667066, ISSN: 1087-0156, DOI: 10.1038/s41587-021-01009-z
TAKEDA SATORU N.; NAKAGAWA RYOYA; OKAZAKI SAE; HIRANO HISATO; KOBAYASHI KAN; KUSAKIZAKO TSUKASA; NISHIZAWA TOMOHIRO; YAMASHITA KEI: "Structure of the miniature type V-F CRISPR-Cas effector enzyme", MOLECULAR CELL, ELSEVIER, AMSTERDAM, NL, vol. 81, no. 3, 16 December 2020 (2020-12-16), AMSTERDAM, NL, pages 558 - 570, XP086487104, ISSN: 1097-2765, DOI: 10.1016/j.molcel.2020.11.035
TAKEDA SATORU N.; NAKAGAWA RYOYA; OKAZAKI SAE; HIRANO HISATO; KOBAYASHI KAN; KUSAKIZAKO TSUKASA; NISHIZAWA TOMOHIRO; YAMASHITA KEI: "Structure of the miniature type V-F CRISPR-Cas effector enzyme", MOLECULAR CELL, ELSEVIER, AMSTERDAM, NL, vol. 81, no. 3, 16 December 2020 (2020-12-16), AMSTERDAM, NL, pages 558 - 570, XP086487104, ISSN: 1097-2765, DOI: 10.1016/j.molcel.2020.11.035
Attorney, Agent or Firm:
SEUM IP (KR)
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