Nonviral vectors, gene therapy products

For gene therapy products, the selection of the vector system is put to the test initially in the POC studies. These studies provide the first in vitro assessment of the choice of the viral or nonviral vector, the cell population to be used (if applicable), as well as the selection of a therapeutic transgene (if applicable) to treat a specific disease. 

Most of today s approved biotechnology products are produced in bacteria, yeast, or mammalian cells. Newer sources currently used to manufacture clinical trial materials include insect cells, transgenic animals, and gene therapy vectors. Other potential sources include transgenic plants and nonviral delivery systems... 

Gene therapy Contrary to antisense therapy, the ability to induce production of specific proteins identified by proteomics has similar potential for future clinical therapeutics. In vitro application is widespread, and researchers continue to develop new liposome [10,49], synthetic [33,34,50-52] and natural polymer [25,27], and peptide [28] vectors in pursuit of more efficient and compatible formulations. While human in vivo application of nonviral gene therapy remains limited, the wealth of research focused on developing suitable vectors cannot sufficiently emphasize the potential for successful application-specific transfection vehicles. 

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