Pseudo-hydrodynamic Delivery of Helper-dependent Adenoviral Vectors into Non-human Primates for Liver-directed Gene Therapy
نویسندگان
چکیده
منابع مشابه
Helper-dependent adenoviral vectors as gene delivery vehicles
Adenoviral (Ad)-mediated in vivo gene transfer and expression is limited in part by cellular immune responses to viralencoded proteins. In an attempt to diminish these responses, we have previously developed and described helperdependent (HD) Ad vectors in which the viral protein coding sequences are completely deleted. These vectors provided efficient delivery, and greater safety which represe...
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In the majority of potential applications gene therapy will require an effective transfer of a transgene in vivo resulting in high-level and long-term transgene expression, all in the absence of significant toxicity or inflammatory responses. The most efficient vehicles for delivery of foreign genes to the target tissues are modified adenoviruses. Adenoviral vectors of the first generation, des...
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Airway gene delivery is a promising strategy to treat patients with life-threatening lung diseases such as cystic fibrosis (CF). However, this strategy has to be evaluated in large animal preclinical studies in order to translate it to human applications. Because of anatomic and physiological similarities between the human and pig lungs, we utilized pig as a large animal model to examine the sa...
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The efficiency of first-generation adenoviral vectors as gene delivery tools is often limited by the short duration of transgene expression, which can be related to immune responses and to toxic effects of viral proteins. In addition, readministration is usually ineffective unless the animals are immunocompromised or a different adenovirus serotype is used. Recently, adenoviral vectors devoid o...
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Recombinant Adenoviral vectors represent one of the best gene transfer platforms due to their ability to efficiently transduce a wide range of quiescent and proliferating cell types from various tissues and species. The activation of an adaptive immune response against the transduced cells is one of the major drawbacks of first generation Adenovirus vectors and has been overcome by the latest g...
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ژورنال
عنوان ژورنال: Molecular Therapy
سال: 2007
ISSN: 1525-0016
DOI: 10.1038/sj.mt.6300102