RNA NANOTECHNOLOGY FOR CANCER THERAPY

Cancer is a leading cause of death worldwide. Currently available therapies are inadequate and improved technologies and therapies are urgently needed.

Conventional chemotherapeutic agents via oral or intravenous drug delivery are distributed indiscriminately throughout the body, leading to unintended adverse effects to healthy tissue. Several liposome and polymer-based nanoparticle delivery systems have recently been developed. However, clinical translation of these prepared nanoparticles still presents several challenges including:

1) lack of efficacy in blocking tumor progression and prevent metastasis,

2) absence of specific cancer targeting,

3) inefficiency of targeted tissue penetration and intracellular delivery,

4) safety issue due to healthy tissue/organ accumulation and nonspecific cell entry, and

5) nanoparticle heterogeneity, aggregation, and dissociation.

Advances in RNA nanotechnology towards the development of nano-RNA therapeutics, the new-generation individualized nanomedicines, hold great promise to improve therapeutic strategies against cancer. We discovered that pRNA-3WJ nanoparticle-based drug delivery systems offer revolutionary opportunities to develop highly effective targeted therapeutics with improved pharmacodynamic, pharmacokinetic, and safety profiles. This novel pRNA-3WJ nanotechnology can overcome conventional cancer therapeutic limitations, provides novel approach for specifically delivering therapeutic siRNA, microRNA, etc. to specific cancer cells without damaging normal cells, reduces the toxicity and side effect, improves the therapeutic effect, and exhibits great potential in clinical cancer therapy. The advantages of the novel pRNA-3WJ nano-therapeutics include:

1) improved (superior) efficacy in blocking tumor progression and prevent metastasis,

2) specific cancer cell targeting,

3) efficient targeted intracellular delivery, and

4) highly safe because there is no damage to normal tissue and cells.