Invention Application
- Patent Title: ANTISENSE OLIGONUCLEOTIDE AND COMPOSITION FOR PREVENTION OR TREATMENT OF GLYCOGEN STORAGE DISEASE TYPE IA
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Application No.: US18473936Application Date: 2023-09-25
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Publication No.: US20250101431A1Publication Date: 2025-03-27
- Inventor: Go Tajima , Satoshi OKADA , Miyuki TSUMURA
- Applicant: NATIONAL CENTER FOR CHILD HEALTH AND DEVELOPMENT , HIROSHIMA UNIVERSITY , DAIICHI SANKYO COMPANY, LIMITED
- Applicant Address: JP Tokyo; JP Hiroshima; JP Tokyo
- Assignee: NATIONAL CENTER FOR CHILD HEALTH AND DEVELOPMENT,HIROSHIMA UNIVERSITY,DAIICHI SANKYO COMPANY, LIMITED
- Current Assignee: NATIONAL CENTER FOR CHILD HEALTH AND DEVELOPMENT,HIROSHIMA UNIVERSITY,DAIICHI SANKYO COMPANY, LIMITED
- Current Assignee Address: JP Tokyo; JP Hiroshima; JP Tokyo
- Priority: JP2017-046766 20170310
- Main IPC: C12N15/113
- IPC: C12N15/113
Abstract:
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.
Information query
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