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