TY  - JOUR
A1  - Kaufmann, Kerstin
A1  - Büning, Hildegard
A1  - Galy, Anne
A1  - Schambach, Axel
A1  - Grez, Manuel
T1  - Gene therapy on the move
T2  - EMBO molecular medicine
N2  - 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.
KW  - clinical trials
KW  - iPS
KW  - monogenic disorders
KW  - stem cell therapy
KW  - viral vectors
Y1  - 2013
UR  - http://publikationen.ub.uni-frankfurt.de/frontdoor/index/index/docId/32140
UR  - https://nbn-resolving.org/urn:nbn:de:hebis:30:3-321405
SN  - 1757-4676
SN  - 1757-4684
N1  - 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.
VL  - 5
IS  - 11
SP  - 1642
EP  - 1661
PB  - Wiley-Blackwell
CY  - Chichester
ER  -