Title:
NOVEL INTRON FRAGMENTS
Document Type and Number:
WIPO Patent Application WO/2022/010277
Kind Code:
A1
Abstract:
Novel intron fragments are provided. The intron fragments can increase gene expression to levels equal to or higher than those achieved by the full-length intron while maintaining their ability to increase gene expression even when combined with various types of promoters and splicing donors. Particularly, the intron fragments enable loading of larger transgenes when used in genetic information delivery systems whose size is limited, for example, adeno-associated viruses (AAVs) and rhabdoviruses. Therefore, the use of the intron fragments is expected to extend the range of therapeutic genes.
Inventors:
KONG HOON YOUNG (KR)
KIM JONG-MOOK (KR)
KIM JEE YONG (KR)
SHIN SUNHWA (KR)
LEE KYUNGWON (KR)
HAN JOO SEOK (KR)
KIM JONG-MOOK (KR)
KIM JEE YONG (KR)
SHIN SUNHWA (KR)
LEE KYUNGWON (KR)
HAN JOO SEOK (KR)
Application Number:
PCT/KR2021/008693
Publication Date:
January 13, 2022
Filing Date:
July 08, 2021
Export Citation:
Assignee:
NEURACLE GENETICS INC (KR)
International Classes:
C12N15/113; A61K39/12; A61P27/02; C12N15/86
Domestic Patent References:
| WO2009139004A2 | 2009-11-19 | |||
| WO2020106916A1 | 2020-05-28 |
Other References:
DATABASE PROTEIN 30 January 2007 (2007-01-30), ANONYMOUS : "Cloning vector pAAV-EF1alpha-hFAH.AOS2, complete sequence", XP055887665, retrieved from GENBANK Database accession no. EF203084
HIROYUKI NAKAI, THERESA A. STORM, MARK A. KAY: "Increasing the size of rAAV-mediated expression cassettes in vivo by intermolecular joining of two complementary vectors", NATURE BIOTECHNOLOGY VOL, 1 January 2000 (2000-01-01), pages 527 - 532, XP055660100, Retrieved from the Internet [retrieved on 20200121], DOI: 10.1038/75390
JIAMIAO LU, JAMES A. WILLIAMS, JEREMY LUKE, FEIJIE ZHANG, KIRK CHU, MARK A. KAY: "A 5' Noncoding Exon Containing Engineered Intron Enhances Transgene Expression from Recombinant AAV Vectors in vivo", HUMAN GENE THERAPY, vol. 28, no. 1, 1 January 2017 (2017-01-01), GB , pages 125 - 134, XP055587331, ISSN: 1043-0342, DOI: 10.1089/hum.2016.140
HIROYUKI NAKAI, THERESA A. STORM, MARK A. KAY: "Increasing the size of rAAV-mediated expression cassettes in vivo by intermolecular joining of two complementary vectors", NATURE BIOTECHNOLOGY VOL, 1 January 2000 (2000-01-01), pages 527 - 532, XP055660100, Retrieved from the Internet
JIAMIAO LU, JAMES A. WILLIAMS, JEREMY LUKE, FEIJIE ZHANG, KIRK CHU, MARK A. KAY: "A 5' Noncoding Exon Containing Engineered Intron Enhances Transgene Expression from Recombinant AAV Vectors in vivo", HUMAN GENE THERAPY, vol. 28, no. 1, 1 January 2017 (2017-01-01), GB , pages 125 - 134, XP055587331, ISSN: 1043-0342, DOI: 10.1089/hum.2016.140
Attorney, Agent or Firm:
CHUNG HYUN PATENT & LAW FIRM (KR)
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