Abstract:
: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.
journal_name
J Viroljournal_title
Journal of virologyauthors
Gao GP,Lu Y,Sun X,Johnston J,Calcedo R,Grant R,Wilson JMdoi
10.1128/JVI.00526-06subject
Has Abstractpub_date
2006-06-01 00:00:00pages
6192-4issue
12eissn
0022-538Xissn
1098-5514pii
80/12/6192journal_volume
80pub_type
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pub_type: 杂志文章
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journal_title:Journal of virology
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journal_title:Journal of virology
pub_type: 杂志文章
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