Highly efficient retinal gene delivery with helper-dependent adenoviral vectors
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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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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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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Helper-dependent adenoviral vectors mediate high efficiency gene editing in induced pluripotent stem cells without needing a designer nuclease thereby avoiding off-target cleavage. Because of their large cloning capacity of 37 kb, helper-dependent adenoviral vectors with long homology arms are used for gene editing. However, this makes vector construction and recombinant analysis difficult. Con...
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ژورنال
عنوان ژورنال: Genes & Diseases
سال: 2014
ISSN: 2352-3042
DOI: 10.1016/j.gendis.2014.09.002