Antisense oligonucleotide and composition for prevention or treatment of glycogen storage disease type Ia

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United States of America Patent

PATENT NO 11963974
APP PUB NO 20200246369A1
SERIAL NO

16491435

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Abstract

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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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Patent Owner(s)

Patent OwnerAddress
HIROSHIMA UNIVERSITY3-2 KAGAMIYAMA 1-CHOME HIGASHI-HIROSHIMA-SHI HIROSHIMA 7398511 ?7398511
DAIICHI SANKYO COMPANY LIMITED3-5-1 NIHONBASHI HONCHO CHUO-KU TOKYO 1038426 ?1038426
NATIONAL CENTER FOR CHILD HEALTH AND DEVELOPMENT2-10-1 OKURA SETAGAYA-KU TOKYO 1578535 ?1578535

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Inventor(s)

Inventor Name Address # of filed Patents Total Citations
Okada, Satoshi Hiroshima, JP 287 3722
Tajima, Go Tokyo, JP 2 1
Tsumura, Miyuki Hiroshima, JP 2 0

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