Session 3: Genome Engineering and Therapeutics
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Owing to the advent of the chimeric antigen receptor (CAR), cell-based immunotherapy has provided a unique avenue to treating oncological malignancies that are currently limited by traditional treatments such as chemotherapy and radiation. More recently, the use of engineered natural killer (NK) cells expressing CARs (CAR-NK) have been shown to possess an improved safety profile compared to CAR-T cells, making CAR-NKs a desirable treatment option for many cancers. However, primary sources ...
The focus of precision oncology over the last 25 years has been on targeting druggable oncogenes with gain-of-function genetic alterations (i.e., BCR-ABL and EGFR). However, most solid tumors do not carry these types of mutations, but instead alterations in undruggable oncogenes or loss-of-function mutations in tumor suppressors. Synthetic lethality is a concept in which the simultaneous targeting of two pathways results in cell death, whereas the targeting of either pathway alone does not...
Genetic screens are one of the most potent unbiased tools to identify gene functions. Following pioneering studies of whole genome gene deletion studies in yeast, CRISPR/Cas9 pooled genome-wide KO screens now allow similar screens to interrogate directly the genetics of human cell lines. When made in the presence of bioactive compounds affecting growth (chemogenomic screens), the genes whose knockout are deleterious to growth (sensitizers or synthetic lethals) or favor growth (rescues or s...