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

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 Virol

journal_title

Journal of virology

authors

Gao GP,Lu Y,Sun X,Johnston J,Calcedo R,Grant R,Wilson JM

doi

10.1128/JVI.00526-06

subject

Has Abstract

pub_date

2006-06-01 00:00:00

pages

6192-4

issue

12

eissn

0022-538X

issn

1098-5514

pii

80/12/6192

journal_volume

80

pub_type

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