Gene therapy, definition

Although many of the results generated to date provide hope for the future, gene therapy thus far has failed to provide a definitive cure for cancer. The lack of success is likely due to a number of factors, including ... 

We have included gene therapy and stem cells to present a more comprehensive perspective on medical treatments, although they are not drugs by our conventional definitions. 

Definitive example of successful gene therapy in two patients suffering from ADA-SCID. More patients need to be enrolled in similar trials to confirm the safety, efficacy and optimization of the therapy, and also with regard to the requirement of nonmyeloablative conditioning. 

The efficacy of gene transfer approaches to therapeutic angiogenesis is now being tested in clinical trials. Controlled phase II trials are providing positive but not definitive results. Gene therapy appears to be safe based on these data. Hard clinical endpoints, such as mortality, myocardial infarction, and the need for revascularization are lacking, as is long-term follow-up. 

Retroviruses are the subject of extensive research in virology these days for several reasons. The first is that retroviruses have been linked to cancer, and more aspects of the relationship between viruses and cancer are discovered every day. The second is that human immimodeficiency virus (HIV) is a retrovirus. HIV is the causative agent of the disease acquired immimodeficiency syndrome (AIDS). The search for AIDS treatments and a definitive cure remains one of the primary goals of retroviral research. HIV will be covered more extensively in the Hot Topics Magazine. The third is that retroviruses can be used in gene therapy, as described in the following Biochemical Connections box. 

Gustafson, J., Greish, K., Frandsen, J. etoL (2009) SUk-elastin-like recombinant polymers for gene therapy of head and neck cancer from molecular definition to controlled gene expression. Journal of Controlled Release, 140,256-261. 

The Introduction will give a brief description of DNA as a biopolymer (structure, conformations, topologies), some definitions in the field of polyelectrolytes (weak and strong polyelectrolytes), some generalities about DNA/ polycation complexes (factors influencing the complexation, models describing the structure of the polyplexes, methods adapted to their characterization), and a description of the parameters to take into consideration for their use in gene therapy. 

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