Full-Length Dystrophin Reconstitution with Adeno-Associated Viral 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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Adeno-associated virus (AAV) is a defective, non-pathogenic human parvovirus that depends for growth on coinfection with a helper adenovirus or herpes virus. Recombinant adeno-associated viruses (rAAVs) have attracted considerable interest as vectors for gene therapy. In contrast to other gene delivery systems, rAAVs lack all viral genes and show long-term gene expression in vivo without immune...
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Neurodegenerative monogenic diseases often affect tissues and organs beyond the nervous system. An effective treatment would require a systemic approach. The intravenous administration of novel therapies is ideal but is hampered by the inability of such drugs to cross the blood-brain barrier (BBB) and precludes efficacy in the central nervous system. A number of these early lethal intractable d...
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ژورنال
عنوان ژورنال: Human Gene Therapy
سال: 2014
ISSN: 1043-0342,1557-7422
DOI: 10.1089/hum.2013.210