منابع مشابه
Syngeneic AAV Pseudo-particles Potentiate Gene Transduction of AAV Vectors
Adeno-associated virus (AAV) vectors have emerged as a safe and efficient gene therapy platform. One complication is that a significant amount of empty particles have always been generated as impurities during AAV vector production. However, the effects of such particles on AAV vector performance remain unclear. Here we systemically evaluated the biological properties of three types of "empty" ...
متن کاملMarker rescue of adeno-associated virus (AAV) capsid mutants: a novel approach for chimeric AAV production.
Marker rescue, the restoration of gene function by replacement of a defective gene with a normal one by recombination, has been utilized to produce novel adeno-associated virus (AAV) vectors. AAV serotype 2 (AAV2) clones containing wild-type terminal repeats, an intact rep gene, and a mutated cap gene, served as the template for marker rescue. When transfected alone in 293 cells, these AAV2 mut...
متن کاملPackaging of human chromosome 19-specific adeno-associated virus (AAV) integration sites in AAV virions during AAV wild-type and recombinant AAV vector production.
Adeno-associated virus type 2 (AAV-2) establishes latency by site-specific integration into a unique locus on human chromosome 19, called AAVS1. During the development of a sensitive real-time PCR assay for site-specific integration, AAV-AAVS1 junctions were reproducibly detected in highly purified AAV wild-type and recombinant AAV vector stocks. A series of controls documented that the junctio...
متن کاملMuscling through AAV immunity.
AAV-1 is one of the most promising vectors for gene delivery to skeletal muscle. In this issue of Blood, Mingozzi and colleagues now demonstrate that AAV-1-mediated gene transfer into human skeletal muscle results in activation of capsid-specific T cells.
متن کاملAAV and Insertional Mutagenesis.
editorial I n this issue of Molecular Therapy, David Rus-sell provides an invited commentary on data presented at the tenth annual meeting of the American Society of Gene Therapy that strongly suggested that adenovirus-associated virus (AAV) vector insertion could in certain models be linked to the development of cancer. 1 A short report describing the data that are the subject of this commenta...
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ژورنال
عنوان ژورنال: Nature Biotechnology
سال: 1999
ISSN: 1087-0156,1546-1696
DOI: 10.1038/6105