The central dogma of life science is the transcription of DNA to RNA and the translation of RNA to proteins. Many nucleic acids are involved in disease mechanisms. Rather than targeting the complex plethora of protein products, the direct targeting of nucleic acids appears as a promising strategy. However, limitations related to selectivity and potency as well as poor pharmokinetic properties have made the efficient development of nucleic acid targeting modalities challenging. I will present innovative strategies to target oncogenic non-coding RNA using ultra large combinatorial libraries. I will also present the rational design of synthetic transcription factors targeting double stranded DNA sequences involved in cancer development.
