I am trying to identify factors that mediate Antisense oligonucleotides (ASOs) uptake and the underlying mechanisms on how ASOs are released from the internalized compartments (i.e. endocytic compartments). To do that, I established a high-throughput CRISPR (clustered regularly interspaced short palindromic repeats) screen during which genes are disrupted on a whole-genomic scale in a reporter cell line, which expresses the reporter system and Cas9. The screen will help us identify genes that are essential for ASO uptake and activity.
Research associate, Cellular/Molecular Biologist. Lunenfeld-Tanenbaum Research Institute, Sinai Health, Toronto Canada