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.

Date of Patent:
Apr. 23, 2024

Filed:

Mar. 09, 2018
Applicants:

National Center for Child Health and Development, Tokyo, JP;

Hiroshima University, Hiroshima, JP;

Daiichi Sankyo Company, Limited, Tokyo, JP;

Inventors:

Go Tajima, Tokyo, JP;

Satoshi Okada, Hiroshima, JP;

Miyuki Tsumura, Hiroshima, JP;

Attorneys:
Int. Cl.
CPC ...
C12N 15/11 (2006.01); A61K 31/7088 (2006.01); A61K 48/00 (2006.01); A61P 3/08 (2006.01); C12N 15/113 (2010.01);
U.S. Cl.
CPC ...
A61K 31/7088 (2013.01); A61K 48/00 (2013.01); A61P 3/08 (2018.01); C12N 15/113 (2013.01); C12Y 301/03009 (2013.01); C12N 2310/11 (2013.01); C12N 2310/3231 (2013.01); C12N 2310/3233 (2013.01); C12N 2320/33 (2013.01);
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.


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