Gene. 2013 Apr 22. pii: S0378-1119(13)00434-4. doi: 10.1016/j.gene.2013.03.137. [Epub ahead of print]
History of gene therapy.
Source
A.I. Virtanen Institute, Dept. of Biotechnology and Molecular Medicine, Univ. of Eastern Finland, Kuopio, Finland.
Abstract
Two decades after the initial gene therapy trials and more than 1700 approved clinical trials worldwide we not only have gained much new information and knowledge regarding gene therapy in general, but also learned to understand the fear that has persisted in the society. Despite the drawbacks gene therapy has faced, also success stories have been reported. Examples for these are the positive recommendation for a gene therapy product (Glybera) by the EMA for approval in the European Union and the positive trials for the treatment of ADA deficiency, SCID-X1 and adrenoleukodystrophy. Nevertheless, there is still a lot to learn and during the course of time more safety data has become available that helps us to develop better gene therapy approaches. Also, with the increased understanding of molecular medicine, we have been able to develop more specific and efficient gene transfer vectors. In this review, we will take a historical view and highlight some of the milestones that had an important impact on the development of gene therapy. We will also discuss briefly the safety and ethical aspects of gene therapy and raise up some concerns that have been connected with gene therapy as a new therapeutic modality.
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