Universität zu Köln

Kaufmann, Kerstin B. ORCID: 0000-0001-5013-6123, Buening, Hildegard, Galy, Anne, Schambach, Axel and Grez, Manuel (2013). Gene therapy on the move. EMBO Mol. Med., 5 (11). S. 1642 - 1662. HOBOKEN: WILEY. ISSN 1757-4684

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Abstract

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.

Item Type:	Journal Article
Creators:	Creators Email ORCID ORCID Put Code Kaufmann, Kerstin B. UNSPECIFIED orcid.org/0000-0001-5013-6123 UNSPECIFIED Buening, Hildegard UNSPECIFIED UNSPECIFIED UNSPECIFIED Galy, Anne UNSPECIFIED UNSPECIFIED UNSPECIFIED Schambach, Axel UNSPECIFIED UNSPECIFIED UNSPECIFIED Grez, Manuel UNSPECIFIED UNSPECIFIED UNSPECIFIED
URN:	urn:nbn:de:hbz:38-472811
DOI:	10.1002/emmm.201202287
Journal or Publication Title:	EMBO Mol. Med.
Volume:	5
Number:	11
Page Range:	S. 1642 - 1662
Date:	2013
Publisher:	WILEY
Place of Publication:	HOBOKEN
ISSN:	1757-4684
Language:	English
Faculty:	Unspecified
Divisions:	Unspecified
Subjects:	no entry
Uncontrolled Keywords:	Keywords Language CHRONIC GRANULOMATOUS-DISEASE; SEVERE COMBINED IMMUNODEFICIENCY; STEM-CELL TRANSPLANTATION; LEBER CONGENITAL AMAUROSIS; BEAUTY TRANSPOSON SYSTEM; WISKOTT-ALDRICH SYNDROME; INTERNET-BASED PATIENT; LENTIVIRAL VECTOR; T-CELLS; RETROVIRAL INTEGRATION Multiple languages Medicine, Research & Experimental Multiple languages
URI:	http://kups.ub.uni-koeln.de/id/eprint/47281