Polymers for DNA Delivery 英文参考文献.docVIP

Molecules 2005, 10, 34-64 molecules ISSN 1420-3049 Review Polymers for DNA Delivery H. Eliyahu , Y. Barenholz and A. J. Domb 1,2 2 1,* 1 2 Department of Medicinal Chemistry and Natural Products, School of Pharmacy, Jerusalem, Israel Laboratory of Membrane and Liposome Research, Department of Biochemistry, The Hebrew University – Hadassah Medical School, Jerusalem, Israel * Author to whom correspondence should be addressed; e-mail: adomb@md.huji.ac.il Received: 5 July 2004 / Accepted: 21 July 2004 / Published: 31 January 2005 Abstract: Nucleic acid delivery has many applications in basic science, biotechnology, agriculture, and medicine. One of the main applications is DNA or RNA delivery for gene therapy purposes. Gene therapy, an approach for treatment or prevention of diseases associated with defective gene expression, involves the insertion of a therapeutic gene into cells, followed by expression and production of the required proteins. This approach enables replacement of damaged genes or expression inhibition of undesired genes. Following two decades of research, there are two major methods for delivery of genes. The first method, considered the dominant approach, utilizes viral vectors and is generally an efficient tool of transfection. Attempts, however, to resolve drawbacks related with viral vectors (e.g., high risk of mutagenicity, immunogenicity, low production yield, limited gene size, etc.), led to the development of an alternative method, which makes use of non-viral vectors. This review describes non-viral gene delivery vectors, termed self-assembled systems, and are based on cationic molecules, which form spontaneous complexes with negatively charged nucleic acids. It introduces the most important cationic polymers used for gene delivery. A transition from in vitro to in vivo gene delivery is also