Liver gene therapy

Key words Cationic liposomes, Lipoplexes, Asialoglycoprotein receptor, Asiaiofetuin, Liver gene therapy, Gene delivery... 

Grimm D, Zhou S, Nakai H, Thomas CF, Storm TA, Fuess S, Matsushita T, AUen J, Surosky R, Lochrie M, Meuse L, McQeUand A, Colosi P, Kay MA. Prechnical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy. Blood 2003 102 2412-2419. 

The simplest nonviral gene transfer system in use for gene therapy is the injection of naked plasmid DNA (pDNA) into local tissues or the systemic circulation (88, 100). Naked DNA systems are composed of a bacterial plasmid that contains the cDNA of a reporter or therapeutic gene under the transcriptional control of various regulatory elements (101, 102). In recent years, work in several laboratories has shown that naked plasmid DNA (pDNA) can be delivered efficiently to cells in vivo either via electroporation, or by intravascular delivery, and has great prospects for basic research and gene therapy (101). Efficient transfection levels have also been obtained on direct application of naked DNA to the liver (103, 104), solid tumours (105), the epidermis (106), and hair follicles (106). 

Other recently developed biomaterials will be used for quite different purposes in tissue engineering such as artificial pancreas and liver, artificial skin, nerve regeneration, gene therapy vascular grafts, cornea replacement and others.3... 

Brunetti-Pierri N, Ng P. 2006. Progress towards the application of helper-dependent adenoviral vectors for liver and lung gene therapy. Curr Opin Mol Ther. 8 446 154. 

Kojima, H., Fujimiya, M., Matsumura, K., Younan, P., Imaeda, H., Maeda, M. and Chan, L. (2003). NeuroD-betacellulin gene therapy induces islet neogenesis in the liver and reverses diabetes in mice. Nat. Med. 9, 596-603. 

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