Comparative analysis of adeno-associated viral vector serotypes 1, 2, 5, 7, and 8 in mouse brain.
作者： Jean-Marc Taymans , Luk H. Vandenberghe , Chris Van Den Haute , Irina Thiry , Christophe M. Deroose
DOI: 10.1089/HUM.2006.178
关键词: Virus 、 Viral vector 、 Transduction (genetics) 、 Tropism 、 Genetic enhancement 、 Green fluorescent protein 、 Biology 、 Virology 、 Central nervous system 、 Recombinant virus
摘要: Recombinant adeno-associated virus serotype 2 (rAAV2) vectors have been shown to deliver genes effectively neurons in the brain, retina, and spinal cord. The characterization of new AAV serotypes revealed different patterns transduction a diverse array tissues (Gao, G., Vandenberghe, L.H., Wilson, J.M. [2005]. Curr. Gene Ther. 5, 285-297). Here, we extensively compare neural tropism human-derived rAAVs (types 2/1, 2, 2/5) with nonhuman primate-derived 2/7 2/8) adult mouse brain. Mice were injected rAAV type 2/5, 2/7, or 2/8 via caudate-putamen substantia nigra. Intrahippocampal injections also performed for rAAV2/7 rAAV2/8. In all regions injected, transduced almost exclusively. Retrograde pseudotypes was observed particular CNS areas. At high titers, comparable brain volumes targeted except rAAV2, which much smaller volumes. A dose-range comparison intrastriatally types highlighted that efficiency, as determined by volume biophotonic imaging green fluorescent protein expression intensity, significantly higher rAAV2/5 compared rAAV2/8 at low whereas three equally well doses. These results demonstrate use efficiency both human- disease modeling their potential gene therapy.
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