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1. (WO2018164275) ANTISENSE OLIGONUCLEOTIDE AND COMPOSITION FOR PREVENTION OR TREATMENT OF GLYCOGEN STORAGE DISEASE TYPE IA

Pub. No.:    WO/2018/164275    International Application No.:    PCT/JP2018/009326
Publication Date: Fri Sep 14 01:59:59 CEST 2018 International Filing Date: Sat Mar 10 00:59:59 CET 2018
IPC: C12N 15/113
A61K 31/7088
A61K 48/00
A61P 3/08
Applicants: NATIONAL CENTER FOR CHILD HEALTH AND DEVELOPMENT
国立研究開発法人国立成育医療研究センター
HIROSHIMA UNIVERSITY
国立大学法人広島大学
Inventors: TAJIMA, Go
但馬 剛
OKADA, Satoshi
岡田 賢
TSUMURA, Miyuki
津村 弥来
Title: ANTISENSE OLIGONUCLEOTIDE AND COMPOSITION FOR PREVENTION OR TREATMENT OF GLYCOGEN STORAGE DISEASE TYPE IA
Abstract:
The purpose of the present invention is to provide a novel antisense oligonucleotide and a composition for the prevention or treatment of glycogen storage disease type Ia. Provided is an antisense oligonucleotide that: hybridizes the pre-mRNA sequence in a region including at least one base from among the base at position 42911000, the base at position 42911004, and the base at position 42911005 of human chromosome 17 base sequence of GRCh38/hg38; and that is capable of suppressing the abnormal splicing of pre-mRNA of c.648G>T variant G6PC.