Priya S. Kishnani,Suhrad G. Banugaria,Dwight D. Koeberl,Sean N. Prater
申请号:
US13696388
公开号:
US08809282B2
申请日:
2011.05.06
申请国别(地区):
US
年份:
2014
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.