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Polymers for DNA Delivery 英文参考文献
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
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