Simon Liang, PhD

Dr. Liang is a tenure-track assistant professor of Medicine, and his research focuses on the applications of CRISPR-mediated genome editing for gene therapy and cancer research. As a graduate student at University of Bern (Switzerland), he investigated drug resistance mechanisms in lung cancer with FGFR1 amplification and KRAS mutation using CRISPR-based knockout screens. This experience provided him with a deep understanding of how CRISPR technology can be leveraged to elucidate drug resistant mechanisms. During his postdoc training at UMASS medical school, he developed an improved prime editor (PE) capable of generating orthotopic liver cancer models and correcting E342K mutations in PiZ mice. To address safety concerns, he developed two technologies: GUIDE-tag (for SpCas9) and PE-tag (for PE). These tools enable genome-wide profiling of off-target sites in mammalian cell lines and adult mouse liver. In addition, he developed and validated a dual adeno-associated virus (AAV) CRISPR platform capable of effective editing in the mouse lung. To reduce immune responses and enable repeated dosing, he identified inhalable delivery lipids for Cas9 mRNA and sgRNA. These lipid nanoparticles efficiently mediate gene editing in lung epithelium.