Current issues in adeno-associated viral vector production
نویسندگان
چکیده
منابع مشابه
P164: Adeno-Associated Viral Vectors in Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (BMD) is an inherited X-link disease. The incidence of this muscle-wasting disease is 1:5000 male live births. Mutation in the gene coding for dystrophin is the main cause of BMD. Most cases of this disease succumb to respiratory and cardiac failure in 3rd to 4th decades. The slow progression of BMD and recent achievement of gene therapies make it as an appropriate c...
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Mesenchymal stem cells (MSCs) have received considerable attention in the emerging field of regenerative medicine. One aspect of MSC research focuses on genetically modifying the cells with the aim of enhancing their regenerative potential. Adeno-associated virus (AAV) holds promise as a vector for human gene therapy, primarily due to its lack of pathogenicity and low risk of insertional mutage...
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In recent years recombinant adeno-associated viral vectors (AAV) have become increasingly valuable for in vivo studies in animals, and are also currently being tested in human clinical trials. Wild-type AAV is a non-pathogenic member of the parvoviridae family and inherently replication-deficient. The broad transduction profile, low immune response as well as the strong and persistent transgene...
متن کامل6 Adeno - Associated Viral Vectors
Adeno-associated virus (AAV) is a non-pathogenic humanDNAviruswith a unique profile of biological properties that have been of interest to molecular virologists for many years (Berns, 1990; Carter, 1990; Carter et al., 1990). Recently,AAV has also attracted interest as a vector for gene transfer (Carter, 1992; Flotte, 1993a; Hermonat andMuzyczka, 1984; Tratschin et al., 1984). In a general sens...
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It has been shown that the adeno-associated virus (AAV) vector can deliver the VEGF gene efficiently into the ischemic mouse myocardium. However, the AAV genomes can be found in extracardiac organs after intramyocardial injection. To limit unwanted VEGF expression in organs other than the heart, we tested the use of the cardiac myosin light chain 2v (MLC-2v) promoter and the hypoxia-response el...
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ژورنال
عنوان ژورنال: Gene Therapy
سال: 2005
ISSN: 0969-7128,1476-5462
DOI: 10.1038/sj.gt.3302615