The RNA Revolution: from Vaccines to Therapeutic Genome Editing
** Lecture will be given in English**
Abstract:
Objectives: Modulating gene expression that can lead to cell death in the case of tumors or to change of expression of desired proteins for protein replacement therapy or for vaccines or editing of cells for knockout or knockin of genes is a major challenge and an unmet medical need.
Methods: We have developed several strategies to target unique cell surface receptors, as well as a very large library of ionizible lipids with two special features: highly immunogenic or less immunogenic (that can be used for repeated doses). In addition, we developed novel mRNA sequences with more potent immunogenicity that can be utilized against bacterial infection.
Results: In this talk, I will share several examples ranging from new therapeutic targets identified for cancer (e.g. CKAP5) to the first bacterial mRNA vaccine and will also share some data on gene editing for cancer therapeutics.
Conclusions: Cell specific delivery of RNA payloads is feasible and represent the next generation milestone in personalized medicine.
