Peptide-based nanoparticles for delivery Of SiRNA: 'Raspberry Flavor'

摘要

Personalized medicine approaches based on different gene therapy settings have gained much attention lately. In order to enforce successful gene therapy, genetic material needs to be delivered into cells. Nucleic acids and their analogues are unable to do so and thus require assistance to reach their site of action residing in the cytoplasm or nucleus. Here we give a short review on recent advancements in cell-penetrating peptide mediated delivery of splice-correcting oligonucleotides. We report on different cell-penetrating peptides applied for vectorization of splice-correcting oligonucleotides using both covalent conjugation and non-covalent nanoparticle formation approach. While covalent conjugation has gained extensive interest, there have also been great advances in non-covalent complex formation.