Universität zu Köln

Schnoedt, Maria and Buening, Hildegard (2017). Improving the Quality of Adeno-Associated Viral Vector Preparations: The Challenge of Product-Related Impurities. Hum. Gene Ther. Methods, 28 (3). S. 101 - 109. NEW ROCHELLE: MARY ANN LIEBERT, INC. ISSN 1946-6544

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Abstract

Adeno-associated viral (AAV) vectors have emerged as one of the most popular gene transfer systems in both research and clinical gene therapy. As AAV vectors are derived from a stealth, nonpathogenic virus and lack active integrase activity, these vectors are frequently applied for in vivo gene therapy of liver, muscle, and other postmitotic tissues. Although long-term transgene expression from AAV vector episomes is reported from these tissues, the episomal nature of AAV-once regarded as disadvantage-has become an attractive feature for gene-editing approaches targeting proliferating cells. In response to the high demand, AAV vector production is receiving special attention. Besides particle yields and biological activity, the most important concern is improving vector purity. The most difficult task in this regard is removal of defective particles, that is, capsids that are either empty or contain DNA other than the full-length vector genomes. Herein, we characterize and discuss these so-called product-related impurities, methods for their detection, as well as strategies to avoid or reduce their formation.

Item Type:	Journal Article
Creators:	Creators Email ORCID ORCID Put Code Schnoedt, Maria UNSPECIFIED UNSPECIFIED UNSPECIFIED Buening, Hildegard UNSPECIFIED UNSPECIFIED UNSPECIFIED
URN:	urn:nbn:de:hbz:38-228764
DOI:	10.1089/hgtb.2016.188
Journal or Publication Title:	Hum. Gene Ther. Methods
Volume:	28
Number:	3
Page Range:	S. 101 - 109
Date:	2017
Publisher:	MARY ANN LIEBERT, INC
Place of Publication:	NEW ROCHELLE
ISSN:	1946-6544
Language:	English
Faculty:	Unspecified
Divisions:	Unspecified
Subjects:	no entry
Uncontrolled Keywords:	Keywords Language ION-EXCHANGE CHROMATOGRAPHY; REFERENCE-STANDARD MATERIAL; HUMAN GENE-THERAPY; VIRUS TYPE-2; WILD-TYPE; HIGH-TITER; IN-VIVO; TRANSGENE EXPRESSION; AAV VECTORS; NONHOMOLOGOUS RECOMBINATION Multiple languages Biotechnology & Applied Microbiology; Genetics & Heredity; Medicine, Research & Experimental Multiple languages
Refereed:	Yes
URI:	http://kups.ub.uni-koeln.de/id/eprint/22876