chapter  7
32 Pages

Chapter Targeted Delivery Systems of Nonviral Nucleic Acid–Based Therapeutics

Recently, various nucleic acid-based therapeutics have emerged as new classes of innovative medicines. One of the central tenets of cell biology is the fact that DNA or RNA nucleotides can modulate the protein expression of cells and tissues in a specic manner. DNA-based modalities

7.1 Introduction .......................................................................................................................... 115 7.1.1 Overview ................................................................................................................... 115 7.1.2 Current Status ........................................................................................................... 117 7.1.3 Delivery Systems ...................................................................................................... 119

7.1.3.1 Viral Vectors .............................................................................................. 120 7.1.3.2 Nonviral Vectors ........................................................................................ 120

7.2 Targeting Strategies .............................................................................................................. 120 7.2.1 Monoclonal Antibody ............................................................................................... 121 7.2.2 Transferrin ................................................................................................................124 7.2.3 RGD Peptide ............................................................................................................. 127 7.2.4 Folate ........................................................................................................................ 128 7.2.5 Sugar ......................................................................................................................... 129

7.2.5.1 Mannose ..................................................................................................... 130 7.2.5.2 Galactose .................................................................................................... 131

7.2.6 Hyaluronic Acid ........................................................................................................ 131 7.2.7 Aptamer .................................................................................................................... 132 7.2.8 Small-Size Chemicals ............................................................................................... 133 7.2.9 Cell-Mediated Targeting ........................................................................................... 134 7.2.10 Stimulus-Triggered Targeting ................................................................................... 136

7.2.10.1 Electroporation ........................................................................................... 136 7.2.10.2 Ultrasound-Triggered Delivery .................................................................. 136 7.2.10.3 Magnetic Field-Guided Delivery .............................................................. 137

7.3 Future Prospects ................................................................................................................... 138 References ...................................................................................................................................... 140

include plasmid DNA (pDNA), antisense oligonucleotides (AS-ON), and DNA aptamers. RNA-based entities include ribozymes, small interfering RNAs (siRNAs), and RNA aptamers.