Executive Summary : | This study aims to develop a novel method to deliver anti-miRNA while overcoming endosomal degradation of gene therapeutics. The research will use a unique adipose homing peptide sequenced aptamer modified lipid to develop lipodendriplex for adipose tissue-directed delivery of anti-miR-425. The in-house developed dendrimeric templated technique and Quality-by-design approach will be applied to prepare a high miRNA loading, RNAse stable, endosomal escape prone formulation for cytosolic delivery and targeted silencing of miRNA-425. The gene delivery and transfection efficiency will be tested in the 3T3-L1 differentiated adipose cell line and in a C57BL/6J high-fat diet obesity mouse model. The expected outcomes include one IPR related to loading, stabilization, and delivery of gene therapeutics, four research papers in ISI journals, and one Ph.D. student as SRF. |