Antisense oligonucleotide and composition for prevention or treatment of glycogen storage disease type Ia
US11963974B2 · kind B2 · utility
0Cited by
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8Claims
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Key dates
| Filing date | Mar 9, 2018 |
| Grant date | Apr 23, 2024 |
| Priority date | — |
| Expiry date | Mar 9, 2038 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2320/33
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
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.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.