RNA and gene editing : strategies based on non-viral vectors

This activity explores the different classes of RNA, their structures and functions. It then presents their uses in gene therapy through strategies such as gene silencing, exon skipping and therapeutic vaccines (immunotherapy).

This activity introduces the main gene-editing tools i.e. ZFN, TALE nucleases, CRISPR-Cas 9, and basic editing. Practical applications, using the examples of hereditary transthyretin amyloidosis, glycogenosis type 1.a, and β-thalassemia, then provide an understanding of the therapeutic applications of these tools.

This activity covers the main stages in the production of non-viral vectors for RNA transfer: their synthesis, formulation in lipid nanoparticles and quality control.