The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.
The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.
Patent No.:
Date of Patent:
Apr. 16, 2024
Filed:
Mar. 05, 2019
Daiichi Sankyo Company, Limited, Tokyo, JP;
Kobe Gakuin Educational Foundation, Hyogo, JP;
Makoto Koizumi, Tokyo, JP;
Yoshiyuki Onishi, Tokyo, JP;
Takeshi Masuda, Tokyo, JP;
Mitsuhiro Iwamoto, Tokyo, JP;
Yukiko Sekiguchi, Tokyo, JP;
Kentaro Ito, Tokyo, JP;
Shinnosuke Tsuji, Tokyo, JP;
Masafumi Matsuo, Hyogo, JP;
Daiichi Sankyo Company, Limited, Tokyo, JP;
Kobe Gakuin Educational Foundation, Hyogo, JP;
Abstract
The present invention establishes a molecular therapy for glycogen storage disease type Ia. The present invention provides an oligonucleotide of 15-30 bases comprising a nucleotide sequence complementary to die cDNA of G6PC gene with c.648G>T mutation, wherein the oligonucleotide comprises a sequence complementary to a region comprising any site between the 82to the 92nucleotide from the 5′ end of exon 5 of the G6PC gene with c.648C>T mutation, a pharmacologically acceptable salt or solvate thereof. Also provided is a pharmaceutical drug comprising the oligonucleotide, a pharmacologically acceptable salt or solvate thereof (e.g., therapeutic drug for glycogen storage disease type Ia).