Yuan Jia

The extraordinary developments in COVID-19 vaccines have demonstrated the promise of mRNA-based gene therapies. Despite their success, considerable challenges remain for the efficient, safe, and selective delivery of genes to particular tissues, organs, and cells in vivo. The design and discovery of synthetic vectors capable of delivering genes, drugs, or probes to specific cells, organs, and tissues in the body is one of the grand challenges in medicine. I have been working on the design, synthesis, and biological evaluation of a new class of gene delivery agents based on cationic amphiphilic oligomers. The design of these materials is inspired by the Charge-Altering Releasable Transporters (CARTs) developed at Stanford, but targets different mechanisms for the intracellular release of the mRNA. Preliminary results on the delivery of mRNA indicate that one of these new classes of gene delivery agents is effective both in cell culture and in live mice.