Antisense oligonucleotides and glycogen storage disease type Ia preventive or therapeutic compositions

摘要

A novel antisense oligonucleotide and a composition for preventing or treating glycogenous disease type Ia are provided. In the present invention, the antisense oligonucleotide is a human chromosome 17 nucleotide sequence of GRCh38 / hg38, and contains at least one base among the 42911000th base, the 42911004th base, and the 42911005th base. Provided is an antisense oligonucleotide which hybridizes to the sequence of the pre-mRNA in the region and has an activity of suppressing abnormal splicing of the pre-mRNA of c.648G> T mutant G6PC.