High-level transgene expression in nonhuman primate liver with novel adeno-associated virus serotypes containing self-complementary genomes

被引：63

作者：

Gao, Guang-Ping ^{[1
]}

Lu, You ^{[1
]}

Sun, Xun ^{[1
]}

Johnston, Julie ^{[1
]}

Calcedo, Roberto ^{[1
]}

Grant, Rebecca ^{[1
]}

Wilson, James M. ^{[1
]}

机构：

[1] Univ Penn, Sch Med, Gene Therapy Program, Dept Pathol & Lab Med, Philadelphia, PA 19104 USA

来源：

JOURNAL OF VIROLOGY | 2006年 / 80卷 / 12期

关键词：

D O I：

10.1128/JVI.00526-06

中图分类号：

Q93 [微生物学];

学科分类号：

071005 ; 100705 ;

摘要：

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.

引用

页码：6192 / 6194

页数：3

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