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XERODERMA PIGMENTOSUM GROUP F THERAPEUTIC AGENT

机译：Xeroderma Pigmentosum组F治疗剂

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摘要

To provide a means for treating xeroderma pigmentosum group F, for which no disease-causing mutation has been identified to date.　The present invention provides: an antisense oligonucleotide having a base sequence capable of hybridizing with a portion of the intron region of an XPF gene and having activity to inhibit abnormal posttranscriptional modification of the XPF gene, or a pharmaceutically acceptable salt of said antisense oligonucleotide; and a therapeutic agent or a therapeutic composition for xeroderma pigmentosum group F containing said antisense oligonucleotide or pharmaceutically acceptable salt thereof as an active ingredient.

机译：为了提供治疗Xeroderma Pigmentosum组F的方法，其迄今为止没有鉴定出致病突变。本发明提供：反义寡核苷酸，其具有能够与XPF基因的内含子区域的一部分杂交并具有抑制XPF基因的异常后修饰的活性，或者所述反义寡核苷酸的药学上可接受的盐;和治疗剂或用于Xeroderma Pigmentosom组F的治疗组合物，其包含所述反义寡核苷酸或其药学上可接受的盐作为活性成分。

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公开/公告号WO2021112106A1

专利类型
公开/公告日2021-06-10

原文格式PDF
申请/专利权人 DAIICHI SANKYO COMPANY LIMITED;NATIONAL UNIVERSITY CORPORATION TOKAI NATIONAL HIGHER EDUCATION AND RESEARCH SYSTEM;
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申请/专利号WO2020JP44768
发明设计人 OGI TOMOO;NAKAZAWA YUKA;KOIZUMI MAKOTO;SHOJI TAKAO;
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申请日2020-12-02
分类号C12N15/113;A61K31/712;A61K31/7125;A61P17;
国家 JP
入库时间 2022-08-24 19:17:32

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