Engineering adeno-associated virus 2 vectors for targeted gene delivery to atherosclerotic lesions
نویسندگان
چکیده
منابع مشابه
Targeted gene delivery to vascular tissue in vivo by tropism-modified adeno-associated virus vectors.
BACKGROUND Gene therapy offers an unprecedented opportunity to treat diverse pathologies. Adeno-associated virus (AAV) is a promising gene delivery vector for cardiovascular disease. However, AAV transduces the liver after systemic administration, reducing its usefulness for therapies targeted at other sites. Because vascular endothelial cells (ECs) are in contact with the bloodstream and are h...
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SUMMARY The unique life cycle of adeno-associated virus (AAV) and its ability to infect both nondividing and dividing cells with persistent expression have made it an attractive vector. An additional attractive feature of the wild-type virus is the lack of apparent pathogenicity. Gene transfer studies using AAV have shown significant progress at the level of animal models; clinical trials have ...
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Adeno-associated virus (AAV) is currently being used in several human gene therapy trials, including one targeting hemophilia B and another targeting cystic fibrosis, and to date has demonstrated persistent expression without inflammation of the target tissue. Some of the unique features that have distinguished AAV from other gene therapy vectors include (i) its ability to transduce both dividi...
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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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The complete sequence of adeno-associated virus type 1 (AAV-1) was defined. Its genome of 4,718 nucleotides demonstrates high homology with those of other AAV serotypes, including AAV-6, which appears to have arisen from homologous recombination between AAV-1 and AAV-2. Analysis of sera from nonhuman and human primates for neutralizing antibodies (NAB) against AAV-1 and AAV-2 revealed the follo...
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ژورنال
عنوان ژورنال: Gene Therapy
سال: 2007
ISSN: 0969-7128,1476-5462
DOI: 10.1038/sj.gt.3303077