The present disclosure relates to modified antisense oligonucleotides. The nucleotides described herein are of 10 to 40 nucleobases and include a targeting sequence complementary to a target region within intron 1 of a pre-mRNA of the human alpha glucosidase (GAA) gene. The target region includes at least one additional nucleobase compared to the targeting sequence, wherein the at least one additional nucleobase has no complementary nucleobase in the targeting sequence, and wherein the at least one additional nucleobase is internal to the target region.本文之揭示內容係關於經修飾的反義寡核苷酸。本文所描述的核苷酸係10至40個核鹼基且包括與人類α葡萄糖苷酶(GAA)基因之前驅mRNA之內含子1內的目標區域互補的靶向序列。該目標區域相較於該靶向序列包括至少一個額外的核鹼基，其中該至少一個額外的核鹼基在該靶向序列中不具有互補的核鹼基，且其中該至少一個額外的核鹼基對於該目標區域而言係內部的。
