系统评价腺相关病毒载体在小鼠模型中肝脏定向基因转移的效果。
Systematic evaluation of AAV vectors for liver directed gene transfer in murine models.
机构信息
Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, Pennsylvania 19104, USA.
出版信息
Mol Ther. 2010 Jan;18(1):118-25. doi: 10.1038/mt.2009.246. Epub 2009 Oct 27.
Abstract
Vectors based on adeno-associated viruses (AAVs) are being evaluated for use in liver-directed gene therapy. Candidates have been preselected on the basis of capsid structure that plays an important role in determining performance profiles. We describe a comprehensive and statistically powered set of mouse studies designed to compare the performance of vectors based on seven novel AAV capsids. The key criteria used to select candidates for successful gene therapy are high level and stable transgene expression in the absence of toxicity. Based on these criteria, the best performing vectors, AAV8, AAVhu.37, and AAVrh.8, will be further evaluated in nonhuman primates (NHPs).
摘要
基于腺相关病毒（AAV）的载体正被评估用于肝脏定向基因治疗。候选物是基于对决定性能特征起重要作用的衣壳结构预先筛选的。我们描述了一套全面且具有统计学效力的小鼠研究，旨在比较基于七种新型 AAV 衣壳的载体的性能。用于选择成功基因治疗候选物的关键标准是在无毒性的情况下高水平和稳定的转基因表达。根据这些标准，表现最好的载体 AAV8、AAVhu.37 和 AAVrh.8 将在非人类灵长类动物（NHP）中进一步评估。
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