The field of RNA therapeutics has emerged as a powerful and transformative area for the treatment of cancer, infectious diseases, and genetic disorders. RNA offers rapid, programmable, specific interventions with minimal side effects. Notable clinical successes include Onpattro (an siRNA therapy for hereditary transthyretin-mediated amyloidosis) which became the first FDA approved siRNA therapy in 2018, as well as Comirnaty and Spikevax (Pfizer and Moderna mRNA vaccines for COVID-19). However, RNA therapeutics face several challenges in vivo including instability, rapid clearance from circulation, off-target effects, and insufficient uptake or endosomal release. Therefore, the development of RNA delivery systems with high efficiency and cell/organ specificity is critically important. Dr. Jinjun Shi’s lab at Brigham and Women’s Hospital is focused on addressing these challenges through the development of advanced lipid nanoparticles (LNPs) for RNA delivery. My research will center on the discovery of novel LNP systems by identifying new lipid molecules, guided by rational design and a high-throughput screening strategy. Techniques will include organic synthesis, existing and modified LNP formulation protocols, transfection assays, dynamic light scattering, fluorescence microscopy, and flow cytometry. Applications of these LNP systems include genetic modulation, cancer treatment, and immune regulation. Given the vast therapeutic potential and clinical relevance of RNA, it follows that the development of next-generation RNA delivery platforms—featuring improved potency, tolerability, specificity, and scalability— is an incredibly important endeavor.
Northeastern University
