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Title:
OLIGODENDROCYTE-SPECIFIC PROMOTER, MIRNA SPECIFIC TO PLP1 GENE, VECTOR INCLUDING SAID PROMOTER AND/OR MIRNA, AND PHARMACEUTICAL COMPOSITION INCLUDING SAID VECTOR
Document Type and Number:
WIPO Patent Application WO/2019/156115
Kind Code:
A1
Abstract:
The purpose of the present invention is to provide a vector, a promoter and miRNA for the same, and a pharmaceutical composition including the vector, whereby expression of the PLP1 gene can be suppressed specifically in oligodendrocytes in order to treat PMD caused by an abnormality in the PLP1 gene. This oligodendrocyte-specific promoter includes a nucleic acid having at least 90% sequence identity with the base sequence represented by SEQ ID NO: 1. This miRNA specific to the PLP1 gene has a pair of base sequences comprising a predetermined antisense sequence and sense sequence.

Inventors:
INOUE Ken (4-1-1 Ogawahigashi-cho, Kodaira-sh, Tokyo 51, 〒1878551, JP)
RI Kou (4-1-1 Ogawahigashi-cho, Kodaira-sh, Tokyo 51, 〒1878551, JP)
OKADA Takashi (1-1-5 Sendagi, Bunkyo-k, Tokyo 02, 〒1138602, JP)
OHKI Yu (LIMITED 3-5-1, Nihonbashi Honcho, Chuo-k, Tokyo 26, 〒1038426, JP)
KOIZUMI Makoto (LIMITED 3-5-1, Nihonbashi Honcho, Chuo-k, Tokyo 26, 〒1038426, JP)
Application Number:
JP2019/004227
Publication Date:
August 15, 2019
Filing Date:
February 06, 2019
Export Citation:
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Assignee:
NATIONAL CENTER OF NEUROLOGY AND PSYCHIATRY (4-1-1, Ogawahigashi-cho Kodaira-sh, Tokyo 51, 〒1878551, JP)
NIPPON MEDICAL SCHOOL FOUNDATION (1-1-5, Sendagi Bunkyo-k, Tokyo 02, 〒1138602, JP)
DAIICHI SANKYO COMPANY, LIMITED (3-5-1, Nihonbashi Honcho Chuo-k, Tokyo 26, 〒1038426, JP)
International Classes:
C12N15/113; A61K31/7088; A61K35/761; A61K48/00; C12N15/861
Other References:
MONOH KATSUMI. ET AL.: "Structure, expression and chromosomal localization of the gene encoding human 2' , 3' -cyclic-nucleotide 3' -phosphodiesterase", GENE, vol. 129, 1993, pages 297 - 301, XP023539059, doi:10.1016/0378-1119(93)90283-9
KAGIAVA ALEXIA ET AL.: "Gene delivery targeted to oligodendrocytes using a lentiviral vector", THE JOURNAL OF GENE MEDICINE, vol. 16, no. 11-12, 12 November 2014 (2014-11-12), pages 364 - 373, XP055629811
WANG ERMING ET AL.: "MicroRNA expression in mouse oligodendrocytes and regulation of proteolipid protein gene expression", J NEUROSCI RES, vol. 90, no. 9, 14 April 2012 (2012-04-14) - September 2012 (2012-09-01), pages 1701 - 1712, XP055629812
INOUE KEN: "Treatment of congenital hypomyelinating leukodystrophy using the gene suppression effect of morpholino", FINAL RESEARCH REPORT OF GRANTS-IN-AID FOR SCIENTIFIC RESEARCH, 10 May 2017 (2017-05-10)
LEONG IVONE UN SAN. ET AL.: "In vivo testing of microRNA-mediated gene knockdown in zebrafish", JOURNAL OF BIOMEDICINE AND BIOTECHNOLOGY, vol. 2012, no. ID 350352, 2012, pages 1 - 7, XP055629816
TANG XINYING. ET AL.: "Hemagglutinin-targeting artificial microRNAs expressed by adenovirus protect mice from different clades of H5N1 infection", MOLECULAR THERAPY - NUCLEIC ACIDS, vol. 5, no. 4, 19 April 2016 (2016-04-19), pages e311, XP055629819
LI HENG ET AL.: "Gene therapy using adeno-associated virus vectors in a Plpl-transgenic mouse model of Pelizaeus-Merzbacher disease", PROGRAMS AND ABSTRACTS OF ANNUAL MEETING OF THE MOLECULAR BIOLOGY SOCIETY OF JAPAN, vol. 41, no. IP-0687, November 2018 (2018-11-01)
Attorney, Agent or Firm:
HASEGAWA Yoshiki et al. (SOEI PATENT AND LAW FIRM, Marunouchi MY PLAZA 9th fl. 1-1, Marunouchi 2-chome, Chiyoda-k, Tokyo 05, 〒1000005, JP)
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