ABSTRACT

RNA-based therapy has become a promising avenue for the treatment of many human diseases. The therapeutic potential of RNAs, including ribozymes, short hairpin RNA, small interfering RNA (siRNA), microRNA, antisense oligonucleotides, and RNA aptamers, has long been extensively studied (Guo 2010; Keefe et al. 2010; Levy-Nissenbaum et al. 2008; Que-Gewirth and Sullenger 2007; Yan and Levy 2009). A major challenge that remains is the systemic delivery of these moieties (siRNA, ribozyme, etc.) to the desired target cell organelles. In this regard, the packaging RNA (pRNA) nanoparticle delivery system pioneered by Dr. Guo, combined with recent advancement in RNA aptamers, provides an ideal method for nanoscale delivery suitable for in vivo targeted delivery (Guo 2010).