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HERO ID
7170575
Reference Type
Journal Article
Title
History of gene therapy
Author(s)
Wirth, T; Parker, N; Ylä-Herttuala, S; ,
Year
2013
Is Peer Reviewed?
Yes
Journal
Gene
ISSN:
0378-1119
EISSN:
1879-0038
Language
English
PMID
23618815
DOI
10.1016/j.gene.2013.03.137
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 concern that has persisted in society. Despite the setbacks gene therapy has faced, success stories have increasingly emerged. 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, our knowledge continues to grow 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 which are now producing clinical results. 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 address some concerns that have been connected with gene therapy as an important therapeutic modality.
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