Progress and prospects: gene therapy for genetic diseases with helper-dependent adenoviral vectors

被引：57

作者：

Brunetti-Pierri, N. ^{[1
]}

Ng, P. ^{[1
]}

机构：

[1] Baylor Coll Med, Dept Mol & Human Genet, Houston, TX 77030 USA

来源：

GENE THERAPY | 2008年 / 15卷 / 08期

关键词：

adenovirus; helper-dependent adenoviral vector; gutless; liver; cystic fibrosis;

D O I：

10.1038/gt.2008.14

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 ; 081704 ;

摘要：

Preclinical studies in small and large animal models using helper-dependent adenoviral vectors (HDAds) have generated promising results for the treatment of genetic diseases. However, clinical translation is complicated by the dose-dependent, capsid-mediated acute toxic response following systemic vector injection. With the advancements in vectorology, a better understanding of vector-mediated toxicity, and improved delivery methods, HDAds may emerge as an important vector for gene therapy of genetic diseases and this report highlights recent progress and prospects in this field.

引用

页码：553 / 560

页数：8

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