Chapter 3 - Application in gene therapy and DNA/RNA vaccines

MARÍA J.LIMERES; MARÍA A. TOSCANINI; TOMAS BRITO DEVOTO; MARÍA F. LADETTO; GUILLERMO R. CASTRO; MARIA L. CUESTAS

Smart Nanomaterials for Bioencapsulation

Elsevier

Año: 2022; p. 39 - 62

A novel approach to therapy and vaccination is the administration of nucleic acids that modulate the expression of certain proteins either to correct the underlying genetic disorder or to induce protective cell-mediated immunity, respectively. The application of DNA/RNA for therapeutic or prophylactic purposes to human diseases has been hampered by poor clinical outcomes. The development of an efficient carrier for genetic-based medicines is considered a crucial factor for the successful application of DNA/RNA for the treatment or prophylaxis of several human diseases.Nanotechnology has emerged as a promising tool in biomedicine research. This chapter aims to provide an overview of the potential role of nanotechnology as a promising nonviral tool for DNA/RNA delivery. Particular attention will be focused on formulation strategies and their applications in gene therapy and DNA/RNA vaccines. Preclinical and clinical studies will be included in this chapter.