Drug delivery system and nucleic acid therapeutics

Recently, there have been significant advances in the field of harnessing exogenous nucleic acids (NA) such as siRNA mRNA and DNA as therapeutic modalities. NA-based therapy is an attractive strategy to efficiently treat various diseases because NA therapeutics could offer numerous advantages compared to the conventional smallmolecule therapies. However, there are several drawbacks to overcome in the application of NA-based therapeutics, including the instability of NAs and lack of safe and stable NA delivery system. Here, we present a novel, modular NA drug-delivery system based on porous solid nanoparticle. Porous nanoparticles have numerous pores inside where various drug modalities including NAs and proteins could be loaded. The NAs loaded inside porous nanoparticles are protected against nuclease-mediated degradation and show superior stability for longer storage in addition to longer half-life in vivo. The drug loaded nanoparticles are readily uptaken by target cells. In this talk, several examples of bio-therapeutics based on the porous nanoparticle will be presented. We expect that this platform could be an important approach for routine implementation of NA medicine to treat or prevent wide variety of diseases in the near future. 

Figure 1. Schematic illustration of DegradaBALL, porous nanoparticle based DDS, for RNA medicine. 

1. H. Shin, S. Park, Y. Yim, J. Kim, C. Choi, C. Won, D.-H. Min, Adv. Ther. 2018, 1, 1800065 

20211111_대학원세미나_민달희 교수.pdf