Priya S. Kishnani,Suhrad G. Banugaria,Dwight D. Koeberl,Sean N. Prater
申请号：
US15438375
公开号：
US20170165316A1
申请日:
2017.02.21
申请国别(地区):
US
年份:
2017
代理人:
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.
