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METHOD OF TREATING PATIENTS UNDERGOING PROTEIN REPLACEMENT THERAPY, GENE REPLACEMENT THERAPY, OR OTHER THERAPEUTIC MODALITIES

机译:进行蛋白质替代治疗,基因替代治疗或其他治疗手段的患者的治疗方法

摘要

The present invention relates, in general, to a method of treating patients undergoing enzyme replacement therapy (ERT) or other therapy involving the administration of a proteinaceous therapeutic agent as well gene replacement therapy with non-viral or viral vectors, or other therapeutic modality or modalities, used alone or in combination, which involve the administration of exogenous substances for potential therapeutic benefit, including, but not limited to DNA vaccines, siRNA, splice-site switching oligomers (SSOs) as well as RNA-based nanoparticles (RNPs) and nanovaccines. The invention further relates to compounds and compositions suitable for use in such methods.
机译:总的来说,本发明涉及一种治疗正在进行酶替代疗法(ERT)或其他疗法的患者的方法,该方法涉及给予蛋白质治疗剂以及用非病毒或病毒载体或其他治疗方式或其他疗法的基因替代疗法。单独使用或组合使用的方式,包括给予潜在治疗益处的外源性物质,包括但不限于DNA疫苗,siRNA,剪接位点转换低聚物(SSO)以及基于RNA的纳米粒子(RNP)和纳米疫苗。本发明进一步涉及适用于此类方法的化合物和组合物。

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