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Chapter category: Antisense

Targeted Gene Delivery: The Role of Peptide Nucleic Acid

Chapter authors:
Kenneth W. Liang, Feng Liu and Leaf Huang

Receptor-mediated endocytosis can be exploited to achieve efficient cell-specific gene delivery. Our laboratory has used two approaches for targeted gene delivery. One uses polycation as a carrier for plasmid DNA and the other uses peptide nucleic acid (PNA) as a carrier. Targeted gene delivery using polycation carriers has been widely utilized with some success. This approach mainly suffers from large particle size and nonspecific interaction with blood components. These drawbacks have limited this type of vector for in vivo applications. Using PNA as a carrier, on the other hand, allows for smaller particle size and less nonspecific interactions. The stability of this vector in the circulation may be a limiting factor. In addition, both types of vector lack mechanisms for endosome escape and nuclear transport. In this chapter, current developments and uses for targeted gene delivery of each approach are reviewed.

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