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ISCHEMIC-LESION-SITE-SPECIFIC GENE THERAPY

机译:局部缺血部位基因治疗

摘要

In one embodiment, the present invention provides an antisense nucleic acid drug capable of modulating the expression of a target transcript at an ischemic site of a subject. In one embodiment, the present invention provides a composition for modulating the expression of a target transcript at an ischemic site of a subject, the composition including a nucleic acid complex obtained by annealing to each other a first nucleic acid strand that includes an antisense oligonucleotide region for a target transcript and a second nucleic acid strand that includes a complementary region complementary to at least part of the first nucleic acid strand, a lipid being bound to the second nucleic acid strand.
机译:在一个实施方案中,本发明提供了一种反义核酸药物,其能够调节对象的缺血部位的靶转录物的表达。在一个实施方案中,本发明提供了用于调节靶标转录物在受试者的缺血部位的表达的组合物,该组合物包括通过使包含反义寡核苷酸区域的第一核酸链彼此退火而获得的核酸复合物。对于靶转录物和第二核酸链,其包括与第一核酸链的至少一部分互补的互补区,脂质结合至第二核酸链。

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