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
This summer research project offers a valuable opportunity to learn more about CRISPR delivery, and I'm excited to get started. Your support is greatly appreciated as it enables me to pursue meaningful work that I'm passionate about. Thank you for your generosity and for making this research possible.