Research Project
7803668
DESCRIPTION (provided by applicant): RNA interference (RNAi), a biological process for modulating gene expression using short interfering RNA (siRNA) molecules, has emerged as one of the most promising technologies for treating a wide range of difficult-to-cure diseases, including liver diseases, cancer, neurodegenerative diseases, etc. However, lack of effective means to deliver siRNA into targeted organs in the human body significantly hinders clinical applications of RNAi technology, particularly in liver therapy. Our proposal is specifically designed to address this critical challenge. Phase I work will focus on development of a patent-pending targeted siRNA delivery technology with real-time optimization capability. Applying the technology, we will optimize siRNA delivery to ex vivo liver tissue and demonstrate its clinical relevance by delivering specific siRNA to functionally knockdown certain therapeutic targets of liver diseases. Successful Phase I development will lead to extensive future animal study to demonstrate the utility of this novel siRNA delivery technology in controlling liver diseases, such as liver cirrhosis and alcoholic liver, which could potentially benefit millions of patients suffering from these formidable liver diseases. PUBLIC HEALTH RELEVANCE: Success in this project will accelerate the progress of transferring advanced siRNA based therapeutic technology into clinical application. It will benefit public health by revolutionizing clinical treatment of a broad range of difficult-to-cure diseases such as cancers, liver diseases, viral infection, and cardiovascular diseases, etc.
