Delivery platforms for gene therapy

The landscape of disease treatment has been revolutionized by gene therapy, as evidenced by the successes of Alnylam's Onpattro, Vertex Pharmaceuticals's Casgevy, and the COVID-19 vaccines developed by BioNtech/Pfizer and Moderna. However, existing gene therapy methods grapple with challenges such as low efficacy, non-specific biodistribution, and issues related to side effects and toxicity, limiting their broader applications. The Gong Lab is dedicated to addressing these shortcomings through the development of combinatory chemistry-based libraries for screening highly effective gene delivery carriers. Additionally, the lab is engaged in establishing deep sequencing techniques for high-throughput in vivo screening to achieve specific tissue/cell targeting. Furthermore, the lab is actively involved in developing multi-omics strategies to investigate the toxicity and side effects associated with current gene delivery approaches, with the ultimate goal of optimizing low-toxicity gene delivery carriers. The knowledge and technologies derived from this research are then applied to the treatment of various human diseases, including cancers, infectious diseases, and metabolic disorders.