THERAPEUTIC AGENT FOR GLYCOGEN STORAGE DISEASE TYPE IA

摘要

The purpose of the present invention is to establish a molecular therapy for glycogen storage disease type Ia. An oligonucleotide consisting of 15-30 bases which has a nucleotide sequence complementary to cDNA of G6PC gene with c.648GT mutation, said oligonucleotide comprising a sequence which is complementary to a region containing any of the 82nd to 92nd sites from the 5' end of exon 5 of the G6PC gene with the c.648GT mutation, or a pharmacologically acceptable salt or solvate of the same. A drug (e.g., a therapeutic agent for glycogen storage disease type Ia) comprising the aforesaid oligonucleotide or a pharmacologically acceptable salt or solvate of the same.