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Title:
COMPOSITION FOR MODIFYING TARGET GENE
Document Type and Number:
WIPO Patent Application WO/2019/131829
Kind Code:
A1
Abstract:
A delivery method is provided which delivers a gene modifying tool which achieves a high gene modification efficiency in cells. This composition, for inducing genetic modification in a target locus in a cell, contains 1) the compound represented by expression (I) or a salt thereof, 2) structural lipids, and 3) guide RNA or DNA containing a sequence encoding this, and/or RNA-guided nuclease, or nucleic acids including a sequence encoding this. In expression (I), n is an integer 2-5, R is a linear C1-5 alkyl group, a linear C7-11 alkenyl group or a linear C11 alkadienyl group, the wavy lines independently represent cis or trans bonds.

Inventors:
HOTTA Akitsu (36-1 Yoshida-honmachi, Sakyo-ku, Kyoto-sh, Kyoto 01, 〒6068501, JP)
IFUKU Masataka (36-1 Yoshida-honmachi, Sakyo-ku, Kyoto-sh, Kyoto 01, 〒6068501, JP)
FUJIMOTO Naoko (36-1 Yoshida-honmachi, Sakyo-ku, Kyoto-sh, Kyoto 01, 〒6068501, JP)
IWABUCHI Kumiko (36-1 Yoshida-honmachi, Sakyo-ku, Kyoto-sh, Kyoto 01, 〒6068501, JP)
KENJO Eriya (26-1 Muraoka-Higashi 2-chome, Fujisawa-sh, Kanagawa 12, 〒2510012, JP)
MAKITA Yukimasa (26-1 Muraoka-Higashi 2-chome, Fujisawa-sh, Kanagawa 12, 〒2510012, JP)
OCHIAI Rumiko (26-1 Muraoka-Higashi 2-chome, Fujisawa-sh, Kanagawa 12, 〒2510012, JP)
Application Number:
JP2018/048034
Publication Date:
July 04, 2019
Filing Date:
December 27, 2018
Export Citation:
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Assignee:
KYOTO UNIVERSITY (36-1, Yoshida-honmachi Sakyo-ku, Kyoto-sh, Kyoto 01, 〒6068501, JP)
TAKEDA PHARMACEUTICAL COMPANY LIMITED (1-1 Doshomachi 4-chome, Chuo-ku Osaka-sh, Osaka 45, 〒5410045, JP)
International Classes:
C12N15/09; A01K67/027; A61K31/7088; A61K38/46; A61K47/18; A61K47/24; A61K47/28; A61K48/00; A61P21/02; A61P43/00; C07K14/435; C12N1/15; C12N1/19; C12N1/21; C12N5/10; C12N15/12
Domestic Patent References:
WO2016197133A12016-12-08
Foreign References:
CN104116643A2014-10-29
Other References:
MAKITA, YUKIMASA ET AL.: "Advances in genome editing technologies for treating muscular dystrophy", CLINICAL CALCIUM, vol. 27, no. 3, 28 February 2017 (2017-02-28), pages 391 - 399
YIN HAO ET AL.: "Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo", NAT. BIOTECHNOL., vol. 34, no. 3, 2016, pages 328 - 333, XP055540393
YIN HAO ET AL.: "Structure-guided chemical modification of guide RNA enables potent non-viral in vivo genome editing", NAT. BIOTECHNOL., vol. 35, no. 12, 13 November 2017 (2017-11-13), pages 1179 - 1187, XP055484407
ZHANG LINGMIN ET AL.: "Lipid nanoparticle-mediated efficient delivery of CRISPR/Cas9 for tumor therapy", NPG ASIA MATERIALS, vol. 9, 27 October 2017 (2017-10-27), pages e441, XP055624557
HIROSAWA, MOE ET AL.: "Cell -type-specific genome editing with a microRNA-responsive CRISPR-Cas9 switch", NUCLEIC ACID RES., vol. 45, no. 13, 19 May 2017 (2017-05-19), pages e118, XP055534718
AKITA HIDETAKA ET AL.: "A Neutral Envelope-Type Nanoparticle Containing pH-Responsive and SS-Cleavable Lipid-Like Material as a Carrier for Plasmid DNA", ADV. HEALTHCARE MATER., vol. 2, 2013, pages 1120 - 1125, XP055209940
Attorney, Agent or Firm:
OHNO Seiji et al. (OHNO & PARTNERS, Marunouchi Kitaguchi Building 21F 6-5, Marunouchi 1-chome, Chiyoda-k, Tokyo 05, 〒1000005, JP)
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