Gene Therapy: Principles and ApplicationsBlankenstein K. Sikora Gene therapy is one of the fastest developing areas in modern medical research. Transcending the classical preclinical and clinical disciplines, it is likely to have far reaching consequences in the practice of medicine, as we enter the next millennium. Currently, there are over 200 seperate active clinical trials with over 2,500 patients entered. These studies involve over 20 countries and include patients with a wide range of diseases, including cancer, HIV infection; cystic fibrosis (CF), haemophilia, diabetes, immune deficiencies, metabolic disorders, ischaemic heart disease and arthritis. Gene therapy can be defined as the deliberate transfer of DNA for therapeutic purposes. There is a further implication that only specific sequences containing rel evant genetic information are used; otherwise, transplantation procedures involving bone marrow, kidney or liver could be considered a form of gene therapy. The con cept of transfer of genetic information as a practical clinical tool arose from the gene-cloning technology, developed during the 1970s. Without the ability to isolate and replicate defined genetic sequences, it would be impossible to produce purified material for clinical use. The drive for the practical application of this technology came from the biotechnology industry with its quest for complex human biomole cules produced by recombinant techniques in bacteria. Within a decade, pharma ceutical-grade insulin, interferon, interleukin 2 and tumour necrosis factor were all involved in clinical trials. The next step was to obtain gene expression in vivo. |
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Acad activity adeno-associated virus adenovirus allogeneic animals antigens antisense antitumor AS-ODN autologous binding Biol Blankenstein c-myb Cancer Res CD4+ CD8+ cDNA cell lines cellular CFTR clones complexes cystic fibrosis cytokine cytotoxic deaminase defect disease dose effect efficient encoding enzyme epitopes ex vivo function gene delivery gene expression gene gun gene marking Gene Ther gene therapy gene transfer gene-modified genetic genome glucocerebrosidase growth HSVtk human immune response immunogenicity Immunol induced infection inhibition kinase leukemia liposomes liver low-dose Cy lung lymphocytes MDR1 mediated melanoma metastatic MHC class mice models molecules mouse mRNA murine mutation Natl normal P-glycoprotein packaging patients peptide peripheral blood Proc progenitor cells protein protocol rAAV receptor recombinant replication retroviral vectors SCID sequence specific studies T-cell T-lymphocytes target cells therapeutic tion tissue toxicity trans transduced transfection transgene treatment tumor antigens vaccine viral Virol viruses vitro