Membrane-active peptides for non-viral gene therapy: making the safest easier.
Non-viral gene therapy uses engineered nanoparticles in the virus size range for the cell-targeted delivery of therapeutic nucleic acids. A diverse range of macromolecules are suitable for constructing such 'artificial viruses'. However, proteins, either man-made or from natural sources, are especially convenient for mimicking the viral functions critical for gene transfer. Cell penetration is a critical step for the delivery of nucleic acids in sufficient amounts and hence for reaching satisfactory transgene expression levels. Membrane-active peptides have shown great promise because of their positive role in cross-membrane transport and intracellular trafficking, and they have been incorporated into different artificial viruses. In this review, we will discuss the biological properties of these peptides together with the newest rational approaches designed to optimize their application.
Ferrer-Miralles
Neus
N
Institute for Biotechnology and Biomedicine and Department of Genetics and Microbiology, Autonomous University of Barcelona, Bellaterra, Barcelona, Spain.
Vázquez
Esther
E
Villaverde
Antonio
A
D000818:Animals; D001692:Biological Transport / Q000187:drug effects; D004247:DNA / Q000737:chemistry / Q000235:genetics / Q000378:metabolism; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D007425:Intracellular Membranes / Q000378:metabolism; D008954:Models, Biological; D010455:Peptides / Q000737:chemistry / Q000494:pharmacology
D010455:Peptides; D004247:DNA