RNA molecules have in the recent years become a novel area of interest in the development of experimental and therapeutic strategies against monogenetic disorders, cancer and infectious diseases. RNAs are unique in their capacity of interacting with three major groups of biological macromolecules: DNAs, RNAs, and proteins. Therefore, RNA therapeutics will expand the range of druggable targets including conventional proteins, and previously undrugged or undruggable transcripts and genes.
The course aims at providing an updated overview of the molecular and cellular mechanisms of current therapeutic/experimental approaches based on RNA drugs.
Students will acquire a comprehensive knowledge of the different types of RNA drugs such as antisense oligonucleotides (ASOs), RNA aptamers, small interfering RNAs, microRNAs, mRNAs, and the guide RNA based CRISPR/Cas9 system used for genome editing. Their mechanism of action will be analyzed together with the challenges concerning their stability, specificity, safety, and delivery.
The laboratory module will allow the students to develop practical skills and test some RNA based drugs in vitro in cell models. A fundamental part of the laboratory is to critically evaluate the experimental design and interpretation and to summarize and expose the obtained data to the classmates and the teacher taking into consideration also scientific literature.