Gene therapy applications

Ferreira, G.N.M., Cabral, J.M.S., and Prazeres, D.M.F., Development of process flow sheets for the purification of supercoiled plasmids for gene therapy applications, Biotechnol. Prog. 15, 725, 1999. 

Lewin, A. and Hauswirth, W. 2001. Ribozyme gene therapy applications for molecular medicine. Trends in Molecular Medicine 7(5), 221-228. 

Wu, N. and Ataai, M. 2000. Production of viral vectors for gene therapy applications. Current Opinion in Biotechnology 11(2), 205-208. 

Lewin, A. S., and W. W. Hauswirth. Ribozyme Gene Therapy Applications for Molecular Medicine. Trends in Molecular Medicine 1 no. 5 (2001) 221-228. 

While short-term, high-level gene expression may be appropriate for some gene therapy applications, it would be of less use for the treatment of, for example, genetic diseases, where long-term gene expression would be required. This could be achieved in theory by repeat administration of the adenoviral vector. However, adenoviruses prompt a strong immune response, which limits the efficacy of repeat administration. 

Lundstrom, K., Alphavirus vectors for gene therapy applications. Curr Gene Ther, 2001.1(1) 19-29. 

Stress inducible promoters are under active consideration and investigation in gene therapy applications, and include heat shock, radiation, hypoxia and metabolite inducible promoters. Each has advantages and disadvantages to be considered when addressing specific applications. 

Most gene therapy applications require extravasation of the DNA carriers so that only relative small DNA complexes can pass through the blood vessels and interact directly with parenchymal cells after vascular administration [2]. Under pathophysiological conditions, the structure of the vasculature can change. This phenomenon - termed the enhanced permeation effect - has been utilized to passively target macromolecules to tumors, since blood vessels in tumors are relatively more leaky. 

Duan, D., Yue, Y., Yan, Z. and Engelhardt, J. F. (2003). Trans-splicing vectors expand the packaging limits of adeno-associated virus for gene therapy applications. Methods Mol. Med. 76, 287-307. 

Support for this work was provided by NIH Grant NS POl 36302 to N. M. and R. J. M. N. M. is an inventor on patents related to recombinant AAV technology and owns equity in a gene therapy company that is commercializing AAV for gene therapy applications. 

Clinical gene therapy applications to interventional cardiology... 

Suzuki M, Matsuse T, Isigatsuho Y. Gene therapy for lung diseases development in the vector biology and novel concepts for gene therapy applications. Curr Mol Med 2001, 1, 67-79. 

Establishment of an environment for expanding a cell population that is later extracted from the scaffold for implementation within the body in a cell-based or gene therapy application. 

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