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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 (JP)
RI KOU (JP)
OKADA TAKASHI (JP)
OHKI YU (JP)
KOIZUMI MAKOTO (JP)
Application Number:
JP2019/004227
Publication Date:
August 15, 2019
Filing Date:
February 06, 2019
Export Citation:
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Assignee:
NAT CENTER NEUROLOGY & PSYCHIATRY (JP)
NIPPON MEDICAL SCHOOL FOUND (JP)
DAIICHI SANKYO CO LTD (JP)
International Classes:
C12N15/113; A61K31/7088; A61K35/761; A61K48/00; C12N15/861
Foreign References:
Other References:
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Attorney, Agent or Firm:
HASEGAWA Yoshiki et al. (JP)
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