Universität zu Köln

Hagedorn, Claudia, Schnoedt-Fuchs, Maria, Boehme, Philip, Abdelrazik, Heba, Lipps, Hans J. and Buening, Hildegard (2017). S/MAR Element Facilitates Episomal Long-Term Persistence of Adeno-Associated Virus Vector Genomes in Proliferating Cells. Hum. Gene Ther., 28 (12). S. 1169 - 1180. NEW ROCHELLE: MARY ANN LIEBERT, INC. ISSN 1557-7422

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Abstract

Adeno- associated virus (AAV) vectors are one of the most frequently applied gene transfer systems in research and human clinical trials. Since AAV vectors do not possess an integrase activity, application is restricted to terminally differentiated tissues if transgene expression is required long term. To overcome this limitation and to generate AAV vectors that persist episomally in dividing cells, AAV vector genomes were equipped with a scaffold/matrix attachment region (S/MAR). After a mild antibiotic selection, cells transduced with AAV- S/MAR established colonies that maintained long- term transgene expression (> 50 population doublings) from replicating AAV vector episomes in the absence of further selection. Unexpectedly, with a lesser but still significant efficiency, the control vector (AAV- DS/MAR), a standard single- stranded AAV vector, also established stable transgene- expressing colonies, most of which were maintained as replicating episomes rather than integrated vector genomes. Thus, based on the result in HeLa cells, it is concluded that AAV vector genomes per se possess the ability to establish episomal maintenance in proliferating cells, a feature that can be enhanced by incorporation of a foreign genomic element such as an S/MAR element.

Item Type:	Journal Article
Creators:	Creators Email ORCID ORCID Put Code Hagedorn, Claudia UNSPECIFIED UNSPECIFIED UNSPECIFIED Schnoedt-Fuchs, Maria UNSPECIFIED UNSPECIFIED UNSPECIFIED Boehme, Philip UNSPECIFIED UNSPECIFIED UNSPECIFIED Abdelrazik, Heba UNSPECIFIED UNSPECIFIED UNSPECIFIED Lipps, Hans J. UNSPECIFIED UNSPECIFIED UNSPECIFIED Buening, Hildegard UNSPECIFIED UNSPECIFIED UNSPECIFIED
URN:	urn:nbn:de:hbz:38-210148
DOI:	10.1089/hum.2017.025
Journal or Publication Title:	Hum. Gene Ther.
Volume:	28
Number:	12
Page Range:	S. 1169 - 1180
Date:	2017
Publisher:	MARY ANN LIEBERT, INC
Place of Publication:	NEW ROCHELLE
ISSN:	1557-7422
Language:	English
Faculty:	Unspecified
Divisions:	Unspecified
Subjects:	no entry
Uncontrolled Keywords:	Keywords Language CHIMERIC ANTIGEN RECEPTOR; MODIFIED T-CELLS; NK CELLS; B-CELLS; LEUKEMIA; CD19; LYMPHOMA; ESCAPE; TUMOR; CD20 Multiple languages Biotechnology & Applied Microbiology; Genetics & Heredity; Medicine, Research & Experimental Multiple languages
Refereed:	Yes
URI:	http://kups.ub.uni-koeln.de/id/eprint/21014