Gene therapy on the move

The first gene therapy clinical trials were initiated more than two decades ago. In the early days, gene therapy shared the fate of many experimental medicine approaches and was impeded by the occurrence of severe side e
The first gene therapy clinical trials were initiated more than two decades ago. In the early days, gene therapy shared the fate of many experimental medicine approaches and was impeded by the occurrence of severe side effects in a few treated patients. The understanding of the molecular and cellular mechanisms leading to treatment- and/or vector-associated setbacks has resulted in the development of highly sophisticated gene transfer tools with improved safety and therapeutic efficacy. Employing these advanced tools, a series of Phase I/II trials were started in the past few years with excellent clinical results and no side effects reported so far. Moreover, highly efficient gene targeting strategies and site-directed gene editing technologies have been developed and applied clinically. With more than 1900 clinical trials to date, gene therapy has moved from a vision to clinical reality. This review focuses on the application of gene therapy for the correction of inherited diseases, the limitations and drawbacks encountered in some of the early clinical trials and the revival of gene therapy as a powerful treatment option for the correction of monogenic disorders.
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Metadaten
Author:Kerstin B. Kaufmann, Hildegard Büning, Anne Galy, Axel Schambach, Manuel Grez
URN:urn:nbn:de:hebis:30:3-321405
DOI:http://dx.doi.org/10.1002/emmm.201202287
ISSN:1757-4676
ISSN:1757-4684
Pubmed Id:http://www.ncbi.nlm.nih.gov/pubmed?term=24106209
Parent Title (English):EMBO molecular medicine
Publisher:Wiley-Blackwell
Place of publication:Chichester
Document Type:Article
Language:English
Date of Publication (online):2013/09/17
Date of first Publication:2013/09/17
Publishing Institution:Universitätsbibliothek Johann Christian Senckenberg
Release Date:2014/05/30
Tag:clinical trials; iPS; monogenic disorders; stem cell therapy; viral vectors
Volume:5
Issue:11
Pagenumber:20
First Page:1642
Last Page:1661
Note:
Copyright © 2013 The Authors. Published by John Wiley and Sons, Ltd on behalf of EMBO. This is an open access article under the terms of the Creative Commons Attribution License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited.
HeBIS PPN:365028371
Institutes:Medizin
Georg-Speyer-Haus
Dewey Decimal Classification:610 Medizin und Gesundheit
Sammlungen:Universitätspublikationen
Licence (German):License LogoCreative Commons - Namensnennung 3.0

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