Gene therapy systemic

The investigators evaluated the safety of their nonviral somatic-cell gene therapy system, which they call transkaryotic implantation, in six patients with severe hemophilia. The procedure involved isolation of dermal fibroblasts from the patients upper arms. The fibroblasts were then transfected with a factor VIII genebearing plasmid. Cells that expressed factor VIII were cloned, propagated, and implanted into the patients abdomens. This technique can be considered as a less invasive form of ex vivo gene therapy. 

Dharmacon Roche-Applied Science New England Biolabs Upstate Cell Signaling Solutions Mims Bio Corporation Mims Bio Corporation Gene Therapy Systems, Inc. IC-VEC... 

Some of Wickstrom s work points to the issue of antisense entry into a cell. At least one company, Gene Therapy Systems, is devoted to this (www. genetherapysystems.com). 

Delivery of gene transfer systems of heat shock proteins (Hsp70),250 antioxidant enzymes (catalase)251 or survival genes (Akt)252 has been associated with myocardial protection against ischemia and reperfusion. A vector gene therapy system has also been developed with a hypoxia response element-incorporated promoter to turn on the gene expression in response to hypoxic signal.253... 

The main advantages of optimized suicide gene therapy systems are as follows ... 

Genelabs Technologies Inc. (Asthma) Genelabs Technologies Inc. (Cancer) Gene Therapy Systems Geneformatics Inc. 

Magari S R, Rivera V M, luliucci J D, et al. (1997). Pharmacologic control of humanized gene therapy system implanted into nude mice. J. Clin. Invest. 100 2865-2872. 

To circumvent this problem, vectors that are based on lentiviruses have been developed. In contrast to prototypic retroviruses, lentiviruses do not require cell division for integration. Gene-therapy vectors have been developed from a broad spectrum of lentiviruses including human immunodeficiency vims (HIV), simian and feline immunodeficiency vims as well as visna/maedi vims. The most widely used lentiviral vector system is based on HIV-1. These vectors can efficiently transduce a broad spectrum of dividing and nondividing cells including neurons, hepatocytes, muscle cells, and hematopoietic stem cells [1,2]. 

Kleimnan et al. 2008). In addition, synthetic siRNAs are also subject to degradation in vivo by nuclease activity. Besides side effects and instability, the efficient and specific delivery of the RNAi indncers to the target cell still requires optimization. Here we snmmarize the cnrrent statns of nncleic acid-based antiviral therapentics. The focns will be on antiviral strategies nsing antisense and RNAi technology. Additionally, antiviral ribozymes and aptamers will be discussed briefly, with a focus on recent studies. Gene therapy approaches and delivery systems are the subject of Chapter 11 of this book. 

Milsom MD, Fairbaim LJ (2004) Protection and selection for gene therapy in the hematopoietic system, J Gene Med 6 133-146... 

Tomlinson, E., and Rolland, A.P., Controllable gene therapy pharmaceutics of nonviral gene delivery systems, Journal of Controlled Release, 1995, 39, 357-372. 

Miller, A.D., The problem with cationic liposome/micelle-based non-viral vector systems for gene therapy, Current Medicinal Chemistry, 2003, 10, 1195-1211. 

SchaffertD, Wagner E (2008) Gene therapy progress and prospects synthetic polymer-based systems. Gene Ther 15 1131-1138... 

Moffatt S, Wiehle S, Cristiano RJ (2006) A multifunctional PEI-based cationic polyplex for enhanced systemic p53-mediated gene therapy. Gene Ther 13 1512-1523... 

Hatakeyama H, Akita H, Kogure K, Oishi M, Nagasaki Y, Kihira Y, Ueno M, Kobayashi H, Kikuchi H, Harashima H (2007) Development of a novel systemic gene delivery system for cancer therapy with a tumor-specific cleavable PEG-lipid. Gene Ther 14 68-77... 

Li SD, Huang L (2006) Gene therapy progress and prospects non-viral gene therapy by systemic delivery. Gene Ther 13 1313-1319... 

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