Emily has managed projects involving the development and application of gene engineering and gene modulation technologies including Dharmacon™ ON-TARGETplus™ siRNA and Accell™ siRNA, as well as the introduction of Dharmacon™ Edit-R™, the first rationally-designed, genomewide library of synthetic CRISPRs for gene disruption. She received a Ph.D. in Biochemistry from the University of Colorado at Boulder on the structure and function of protein:single-stranded DNA interactions at telomeres. She has worked extensively on the mechanism, function, specificity, and delivery of RNAi as well as its applications. She is an inventor on many issued patents and has authored peer-reviewed publications in the fields of both RNAi and CRISPR.
Pin-point™ – Modular Base Editing for Clinical Applications
Pin-point™ uses aptameric guide RNA for deaminase recruitment. We demonstrate in CAR-T cells multitargeting of several therapeutically relevant loci, retaining proliferative and cytotoxic ability, including advantages over Cas9. Aptameric recruitment enables simultaneous knock-in and knockout and allows targeting polygenic disorders.
Vanessa Soros, Graphite Bio