NATIONAL CENTER FOR CHILD HEALTH AND DEVELOPMENT;HIROSHIMA UNIVERSITY;DAIICHI SANKYO COMPANY, LIMITED
发明人:
Go TAJIMA,Satoshi OKADA,Myyuki TSUMURA
申请号：
US16491435
公开号：
US20200246369A1
申请日:
2018.03.09
申请国别(地区):
US
年份:
2020
代理人:
摘要：
The present invention provides a novel antisense oligonucleotide and a composition for preventing or treating glycogen storage disease type Ia. The present invention provides an antisense oligonucleotide which hybridizes with a pre-mRNA sequence derived from a region including at least one of a base at position 42911000, a base at position 42911004, and a base at position 42911005 in a base sequence of human chromosome 17 of GRCh38/hg38 and has activity to inhibit aberrant splicing of pre-mRNA of c.648G>；T variant G6PC.
