(203b) Effective RNA Delivery using Janus Base Nanopieces against Cartilage Diseases

Janus base nanotubes (JBNts), a novel family of non-covalent nanomaterials, are self-assembled from Janus bases biomimicking DNA base pairs. Due to their versatile and biomimetic nature, we can engineer these nanotubes into a variety of non-covalent structures and devices for biomedical applications. My research focuses on developing JBNts into tiny, rod-shaped RNA delivery vehicles, named Janus base nanopieces (JBNps). We assembled JBNps by “sandwiching” negative charged delivery cargos (such as RNA) among JBNts. Different from conventional spherical vehicles, JBNps have a superior ability to penetrate tissue matrix barriers and deliver RNA cargos into “difficult-for-drug-delivery” tissues, such as articular cartilage and brain. As a proof-of-concept example, we have used JBNps to successfully deliver therapeutic RNA into cartilage for osteoarthritis treatment in a preclinical animal model.