Polymer Nanoparticles for Gene Delivery: Synthesis and Processing


Kam W. Leong Department of Biomedical Engineering, Johns Hopkins University School of Medicine

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Gene therapy, a therapeutic modality designed to introduce an extraneous gene into the patient's cells to produce a specific protein, potentially can treat a variety of inherited and acquired diseases. Gene medicines are composed of a gene expression vector that encodes a therapeutic protein and a gene delivery system that delivers the gene to the desired tissue and subcellular compartments. The specific and efficient delivery of DNA to the diseased sites and to the nuclei of particular cell populations is the key for successful gene therapy. Gene delivery system plays a central role in fulfilling this mission.