Tissue Specific Delivery of Interfering RNA

Technology

Sangeeta Bhatia and colleagues have developed a method that addresses this problem, with a modular delivery system that allows for predictive targeting of siRNA to tissues of interest in a highly specific manner. Their approach involves the non-covalent attachment of a homing peptide, which binds to a specific cell surface receptor, and protein transduction domain, which facilitates cellular entry, to the siRNA. Unlike many other previously described tissue-specific delivery approaches, components in this method can be rationally designed in a predictive manner, and therefore be easily adapted to many cell types. Furthermore, the components are non-cytotoxic and do not provoke an immune response from the host.

Problem Addressed

Recently, siRNA has shown tremendous promise in treating cancer by suppressing certain genes in tumors. However, delivery is extremely challenging, because siRNA must penetrate the tumor cell to be effective and must be delivered in a targeted fashion to prevent deleterious side-effects.

Advantages

• Modular—can easily be modified to target any tissue of interest
• Enables rational design of future nanomaterials for siRNA delivery
• Noncytotoxic—doesn’t need to be manufactured as a biologic
• Potent, selective inhibition of targets
• Nonstimulatory—no immune response• Can be modified to deliver other oligomers