Résumé
Gene therapy offers the ability to skip, repair or silence defective genes or stimulate the immune system to fight disease by delivering therapeutic nucleic acids (NAs) to patients. Relative to other drugs or protein therapies, NA‐based treatments have the benefit of being a more universal approach to biomedical research due to the polyvalence of NA design. NAs have great potential for therapeutic applications across an immense range of indications. However, the delivery of these exogenous NAs remains difficult and requires a specific delivery system such as viral‐based or nonviral vectors (e.g. lipids, polymers, or peptides). In this context, cell‐penetrating peptides (CPPs) gain more and more interest as NA delivery systems as demonstrated in both cell culture and animal models. Two main strategies have been used to transport NAs with CPPs: covalent conjugation and nanoparticle formation‐based approach.
In this book chapter, we attempt to provide a brief overview of the latest advances in NA delivery with CPPs including their characteristics, their internalization mechanism, examples of NA delivery in vitro and in vivo as well as their functionalization for a more specific delivery.