Gene therapy—the transference of genetic material into an individual—was first conceived as an approach to hereditary single-gene disease. Today the subject of gene therapy comprises multifactorial disorders such as cancer, cardiovascular disease, neurodegenerative disorders, and infectious disease. Ideally, a vector system for gene therapy would have the following attributes: (1) allow efficient transduction of the transgene into the target cells, (2) be safe (i.e., toxicity associated with the vector would be minimal or absent), (3) target only the desired cells within the target tissue, (4) express a therapeutic regulatable amount of the transgene, and (5) not integrate into the host genome. The ideal vector system, however, does not currently exist.
Viral-based vectors are the most common gene delivery systems employed for preclinical or clinical applications and adenoviral (Ad) vectors closely follow retroviruses as the most frequently used vectors for gene therapy. Although Ad vectors are not suitable for all applications, they are very efficient in delivering the therapeutic transgene to the cell nucleus. Other advantages of using Ad vectors include the simplicity of vector construction methods, efficient production, high yields and high stability, and reliable transduction of both proliferating and quiescent cell types.
KeywordsGene Therapy Major Histocompatibility Complex Class Cystic Fibrosis Transmembrane Conductance Regulator Adenoviral Vector Fabry Disease
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