Heidi Huang L&S Biological Sciences
Optimizing CRISPR RNP Formulations for Enhanced Brain Genome Editing
CRISPR-Cas holds great promise for treating Huntington’s Disease, a fatal neurodegenerative disorder. Despite Cas9 RNP’s minimal off-target effects and transient activity, existing RNP delivery methods fall short, limiting RNP therapeutics’ in vivo applications. To address this, the Wilson lab has developed a reversible linker for enhanced RNP brain distribution and editing efficiency. Building on this foundation, my project aims to reduce cytotoxic peptide aggregate formation, which impedes editing efficiency, in Cas9 RNP buffers.
Specifically, my research seeks to optimize CRISPR RNP buffer formulations with selected additives to minimize aggregate formation, aiming for small, uniformly-sized particles that can safely and effectively reach brain tissue. Promising in vitro formulations, as determined in SH-SY5Y5 neuroblastoma cells, will proceed to in vivo testing in mice, providing valuable insights for future clinical applications.
Message To Sponsor
I am incredibly grateful for the support you provided through the SURF fellowship, which has been a transformative experience in my academic journey. This opportunity allowed me to delve deeply into my research on optimizing CRISPR-Cas9 treatments, enhancing my confidence and skills as a researcher. The experience has solidified my aspiration to pursue a career as a physician-scientist working on cell and gene therapies, and it has also fostered a deeper sense of purpose in contributing to the advancement of medical science. Your generosity has not only impacted my scholarly growth but also my personal development, and I am deeply appreciative of your support.