Antisense oligonucleotide and composition for prevention or treatment of glycogen storage disease type ia
AssigneesNational Center for Child Health and Development, Hiroshima University, Daiichi Sankyo Company, Limited
InventorsGo Tajima, Satoshi OKADA, Miyuki TSUMURA
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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.
Claims6
An antisense oligonucleotide that hybridizes with a pre-mRNA sequence of c.648G>T variant G6PC at a region including at least one base corresponding to a base selected from the group consisting of a base at position 648, a base at position 652, and a base at position 653 of a base sequence of c.648G>T variant G6PC, the base sequence being a base sequence shown in SEQ ID NO:
- 4 in which guanine (G) at position 648 is substituted by thymine (T), and that has activity to inhibit aberrant splicing of pre-mRNA of c.648G>T variant G6PC.
Dependencies are inferred from the claim text.