Gene therapy strategy for

Stem CeU-vs, T Cell-Based Gene Therapy Strategies for HIV-Infection. 278... 

Demoly, P. et al. (1997). Gene therapy strategies for asthma. Gene Therapy 4(6), 507-516. 

Raizada MK, Der-Sarkissian S. 2006. Potential of gene therapy strategy for the treatment of hypertension. Hypertension. 47 6-9. 

Castro, M.G., Cowen, R., Smith-Arica, J., Williams, J., Ali, S., Windeatt, S. et al. (2000) Gene therapy strategies for intracranial tumours glioma and pituitary adenomas. Histol. Histopathol., 15, 1233-1252. 

The large number and types of gene therapy trials for cancers reflect both the potential opportunities to develop treatments and the fact that a single strategy will not likely work for aU forms of cancer. 

Insulin-dependent diabetes mellitus (IDDM) is an example of a metabolic disease under active consideration for inducible gene therapy strategies. In this disorder, inflammatory cytokines have been shown to activate apoptosis in pancreatic beta cells. Experimental studies indicate that expression of insulinlike growth factor-1 (IGF-1) can prevent the cytokine-mediated destruction of beta cells of the pancreas (Giannoukakis et al., 2001). Regulated expression of IGF-1 in human pancreatic islets, to preserve beta cell function, may be a useful approach in the treatment of certain types of diabetes (Demeterco and Levine, 2001). 

Dean, D.A. (2000) Peptide nucleic acids versatile tools for gene therapy strategies. Adv. Drug Deliv. Rev., 44, 81-95. 

Two main gene therapy strategies have been intensively studied for the preven-tion of autoimmune diabetes. One is to suppress the expression of autoantigen in islet / -cells, and the other is to use immune suppression cytokines. Glutamic acid decarboxylase (GAD) has been shown to be the most important autoantigen and thus suppression of the GAD expression can treat diabetes (Yoon et al., 1999). For cytokine gene therapy, IL-4 and IL-10 have been the most widely explored. 

The potential success of any gene therapy strategy is, therefore, dependent upon the state of scientific knowledge as to the necessary site of action of the transgene product and on its ability to be transported between compartments. Since the permissiveness to rAAV-mediated gene transfer varies widely between different cell types within different compartments, this initial analysis can greatly affect the potential for success. In the recent years, with the availability of new rAAV serotypes and capsid mutants, this might also dictate the choice of the variety of rAAV vector used. 

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