Title:
COMPOSITION AND METHOD FOR TREATMENT OF LCA10 USING RNA-GUIDED NUCLEASE
Document Type and Number:
WIPO Patent Application WO/2022/240262
Kind Code:
A1
Abstract:
The present invention relates to LCA10 treatment using a CRISPR/Cas12f1(Cas14a1) system. In particular, the present invention relates to a composition comprising a CRISPR/Cas12f1(Cas14a1) system for LCA10 treatment, and to a treatment method using same. In addition, the present invention relates to a composition comprising a CRISPR/Cas12f1(Cas14a1) system for artificially manipulating the CEP290 gene, and to a method of editing the CEP290 gene.
Inventors:
KIM YONG-SAM (KR)
KIM DO YON (KR)
PARK SEYEON (KR)
KIM DO YON (KR)
PARK SEYEON (KR)
Application Number:
PCT/KR2022/006940
Publication Date:
November 17, 2022
Filing Date:
May 13, 2022
Export Citation:
Assignee:
GENKORE CO LTD (KR)
KOREA RES INST BIOSCIENCE & BIOTECHNOLOGY (KR)
KOREA RES INST BIOSCIENCE & BIOTECHNOLOGY (KR)
International Classes:
C12N15/10; C12N9/22; C12N15/113; C12N15/63
Foreign References:
| KR20210053228A | 2021-05-11 | |||
| KR20190058358A | 2019-05-29 | |||
| US20200172886A1 | 2020-06-04 | |||
| CN111363763A | 2020-07-03 | |||
| KR20210051552A | 2021-05-10 | |||
| KR20210050093A | 2021-05-07 | |||
| KR20210044152A | 2021-04-22 | |||
| KR2020014961W | 2020-10-29 | |||
| US20200172886A1 | 2020-06-04 | |||
| KR20210181875A |
Other References:
BIN MOON SU, LEE JEONG MI, KANG JEONG GU, LEE NAN-EE, HA DAE-IN, KIM DO YON, KIM SUN HEE, YOO KWANGSUN, KIM DAESIK, KO JEONG-HEON,: "Highly efficient genome editing by CRISPR-Cpf1 using CRISPR RNA with a uridinylate-rich 3′-overhang", NATURE COMMUNICATIONS, vol. 9, no. 1, 1 December 2018 (2018-12-01), XP055785455, DOI: 10.1038/s41467-018-06129-w
LUCAS B. HARRINGTON, DAVID BURSTEIN, JANICE S. CHEN, DAVID PAEZ-ESPINO, ENBO MA, ISAAC P. WITTE, JOSHUA C. COFSKY, NIKOS C. KYRPID: "Programmed DNA destruction by miniature CRISPR-Cas14 enzymes", SCIENCE, AMERICAN ASSOCIATION FOR THE ADVANCEMENT OF SCIENCE, US, vol. 362, no. 6416, 16 November 2018 (2018-11-16), US , pages 839 - 842, XP055614750, ISSN: 0036-8075, DOI: 10.1126/science.aav4294
MORGAN L. MAEDER ET AL., NATURE MEDICINE, vol. 25, 2019, pages 229 - 233
HARRINGTON ET AL., SCIENCE, vol. 362, 2018, pages 839 - 842
SATORU N. TAKEDA ET AL., MOLECULAR CELL, vol. 81, 2021, pages 1 - 13
MIYAGISHI ET AL., NATURE BIOTECHNOLOGY, vol. 20, 2002, pages 497 - 500
XIA ET AL., NUCLEIC ACIDS RES., vol. 31, no. 17, 1 September 2003 (2003-09-01)
PANYAM ET AL., ADV DRUG DELIV REV, 13 September 2012 (2012-09-13), pages 0169 - 409
KASPAREKHUMPHREY, SEMINARS IN CELL & DEV. BIOL., vol. 22, 2011, pages 886 - 897
LUCAS B. HARRINGTON, DAVID BURSTEIN, JANICE S. CHEN, DAVID PAEZ-ESPINO, ENBO MA, ISAAC P. WITTE, JOSHUA C. COFSKY, NIKOS C. KYRPID: "Programmed DNA destruction by miniature CRISPR-Cas14 enzymes", SCIENCE, AMERICAN ASSOCIATION FOR THE ADVANCEMENT OF SCIENCE, US, vol. 362, no. 6416, 16 November 2018 (2018-11-16), US , pages 839 - 842, XP055614750, ISSN: 0036-8075, DOI: 10.1126/science.aav4294
MORGAN L. MAEDER ET AL., NATURE MEDICINE, vol. 25, 2019, pages 229 - 233
HARRINGTON ET AL., SCIENCE, vol. 362, 2018, pages 839 - 842
SATORU N. TAKEDA ET AL., MOLECULAR CELL, vol. 81, 2021, pages 1 - 13
MIYAGISHI ET AL., NATURE BIOTECHNOLOGY, vol. 20, 2002, pages 497 - 500
XIA ET AL., NUCLEIC ACIDS RES., vol. 31, no. 17, 1 September 2003 (2003-09-01)
PANYAM ET AL., ADV DRUG DELIV REV, 13 September 2012 (2012-09-13), pages 0169 - 409
KASPAREKHUMPHREY, SEMINARS IN CELL & DEV. BIOL., vol. 22, 2011, pages 886 - 897
Attorney, Agent or Firm:
IPLAY PATENT LAW FIRM (KR)
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