Gene therapy with helper-dependent adenoviral vectors: lessons from studies in large animal models

被引:22
作者
Brunetti-Pierri, Nicola [1 ,2 ]
Ng, Philip [3 ]
机构
[1] Telethon Inst Genet & Med, Pozzuoli, Italy
[2] Univ Naples Federico II, Dept Translat Med, Naples, Italy
[3] Baylor Coll Med, Dept Mol & Human Genet, Houston, TX 77030 USA
关键词
Adenovirus; Helper-dependent; Vector; Large animal; Gene therapy; CANINE HEMOPHILIA-B; NONHUMAN-PRIMATES; LONG-TERM; TRANSGENE EXPRESSION; PHENOTYPIC CORRECTION; HEPATIC TRANSDUCTION; CATHETER DELIVERY; IN-VIVO; LIVER; TOXICITY;
D O I
10.1007/s11262-017-1471-x
中图分类号
Q3 [遗传学];
学科分类号
071007 ; 090102 ;
摘要
Helper-dependent adenoviral vectors (HDAd) are deleted of all viral genes and they can efficiently transduce a wide variety of dividing and non-dividing cells to mediate high transgene expression levels. Unlike early generation adenoviral vectors, the absence of viral genes in HDAd results in long-term transgene expression without chronic toxicity and permits a large cloning capacity of 36 kb. Moreover, HDAd genomes exist extra-chromosomally thus minimizing the risks of germline transmission and insertional mutagenesis. For these reasons, HDAd offers tremendous potential for in vivo gene therapy. This chapter reviews preclinical studies using HDAd in large animal models to assess safety and efficacy in a wide variety of gene therapy applications.
引用
收藏
页码:684 / 691
页数:8
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