Inventors:
Priya S. Kishnani - Durham NC, US
Suhrad G. Banugaria - Durham NC, US
Dwight D. Koeberl - Durham NC, US
Sean N. Prater - Durham NC, US
International Classification:
A61K 31/69, A61P 43/00, A61K 39/395
Abstract:
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.