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1546-2080 Online :: 1546-203X Print
Systemic Delivery of siRNA Using Nanoparticles: First Clinical Demonstration of an RNAi Mechanism of Action
Volume 7, Issue 2

Dirk Haussecker

Therapeutics based on RNA interference (RNAi) gene silencing represent a potentially revolutionary approach to drug development. To realize its broad potential, however, researcher must overcome the significant challenge posed by the delivery of the RNAi trigger (siRNA) to the target. While progress has been steady, as reflected in a range of pre-clinical animal models, clinical data is only starting to emerge, and proof-of-concept for RNAi gene silencing following systemic administration is yet to be obtained. In a recent publication, researchers discussed findings that nanoparticles of siRNAs formulated in cyclodextrin-containing polycations (RONDEL) successfully trafficked to cancer tissues in patients following systemic intravenous administration and triggered the degradation of the targeted cancer-related gene RRM2. This marks an important milestone in the clinical development of RNAi therapeutics and provides the rationale for the further development of RONDEL siRNA delivery for cancer and potentially other indications.

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