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Development of gene therapy

Since the late 1980s, gene therapy, more than virtually any other type of therapy, has given rise not only to high expectations of treatment success but also great concerns regarding health risks. Since the Deutsche Forschungsgemeinschaft (German Research Foundation, DFG) issued its first memorandum in 1995, this field of research has developed enormously.

Wide-ranging experiments have shown the therapeutic potential as well as the risks of gene therapy. The second memorandum, which has just been presented by the DFG Senate Commission on Genetic Research, makes it clear that gene therapy already shows signs of success in certain areas, such as severe hereditary immunodeficiency diseases, while in other areas there is still considerable need for research. Moreover, the clinical application of gene therapy requires careful risk/benefit assessment, although in this respect it does not differ substantially from other therapeutic approaches.

Gene therapy is defined as the introduction of genes into tissues or cells via gene transfer, with the purpose of deriving a therapeutic or preventative benefit from the function of these genes. Gene transfer is achieved using a vector ? a kind of vehicle that carries the gene. Somatic gene transfer only targets body cells (somatic cells). Introducing genes into the germline is illegal in Germany. The first well-documented gene therapy studies were launched in the early 1990s. By 2005 it was estimated that more than 1,100 gene therapy studies had been conducted worldwide, one-third of them in Europe, and a significant number in Germany. The DFG is currently funding a Priority Programme for research groups, some of which are international leaders in their field, that examine the entry and persistence of gene therapy vectors in target cells.

In spite of continued great difficulties with technical implementation, initial successes in somatic gene therapy are on the horizon, for example in treating adenosine deam
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Source:Deutsche Forschungsgemeinschaft


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