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Ia THERAPEUTIC AGENT FOR GLYCOGEN STORAGE DISEASE TYPE IA
Ia THERAPEUTIC AGENT FOR GLYCOGEN STORAGE DISEASE TYPE IA
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机译：IA治疗剂糖原储存型IA型
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摘要
Establish molecular therapy for glycogen disease type Ia. An oligonucleotide of 15 to 30 bases consisting of a nucleotide sequence complementary to the cDNA of the G6PC gene having the c.648G>T mutation, from the 82nd to the 92nd from the 5' end of exon 5 of the G6PC gene having the c.648G>T mutation. The oligonucleotide, a pharmacologically acceptable salt or solvate thereof, comprising a sequence complementary to a region containing any one of the first sites. A pharmaceutical comprising the oligonucleotide, a pharmaceutically acceptable salt or solvate thereof (eg, a therapeutic agent for glycogen disease type Ia).
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