High-level transgene expression in nonhuman primate liver with novel adeno-associated virus serotypes containing self-complementary genomes
High-level transgene expression in nonhuman primate liver with novel adeno-associated virus serotypes containing self-complementary genomes
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DOI：
10.1128/jvi.00526-06
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发表时间：
2006-06-01
影响因子：
5.4
通讯作者：
Wilson, James M.
中科院分区：
文献类型：
--
作者：
Gao, Guang-Ping;Lu, You;Wilson, James M.
腺相关病毒（AAV）载体被考虑用于在治疗多种病症中的基因疗法的体内应用。本研究评估了基于新型血清型AAV7和AAV8并含有非人灵长类动物肝脏中的自我互补基因组的第二代载体的生物学。在食蟹猴中实现了稳定水平的转基因表达，并且表明效率比使用传统单链基因组的AAV 2载体可实现的效率高至少2个对数。对组织DNA的分析显示，肝脏中的载体水平很高，似乎与转基因表达的相对量成比例。
Adeno-associated virus (AAV) vectors are being considered for in vivo applications of gene therapy in the treatment of a variety of disorders. This study evaluates the biology of second-generation vectors based on the novel serotypes AAV7 and AAV8 and containing self-complementary genomes in the nonhuman primate liver. Stable levels of transgene expression were achieved in cynomolgus macaques and suggest efficiencies at least 2 log higher than what could be achieved with AAV2 vectors using traditional single-stranded genomes. Analysis of DNAs from tissues revealed high levels of vector in the liver that appeared proportional to the relative amounts of transgene expression.