Expanding Adeno-associated Viral Vector Capacity: A Tale of Two Vectors
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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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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Intracranial injection of viral vectors engineered to express a fluorescent protein is a versatile labeling technique for visualization of specific subsets of cells in different brain regions both in vivo and in brain sections. Unlike the injection of fluorescent dyes, viral labeling offers targeting of individual cell types and is less expensive and time consuming than establishing transgenic ...
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Gene therapy constitutes a therapeutic intervention based on modification of the genetic material of living cells, by correcting genetic defects or overexpressing therapeutic proteins. The success of gene therapy protocols depends on the availability of therapeutically suitable genes, appropriate gene delivery systems and proof of safety and efficacy. Recent advances on the development of gene ...
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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...
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ژورنال
عنوان ژورنال: Biotechnology and Genetic Engineering Reviews
سال: 2007
ISSN: 0264-8725,2046-5556
DOI: 10.1080/02648725.2007.10648098