期刊
MOLECULAR THERAPY
卷 8, 期 1, 页码 151-157出版社
ACADEMIC PRESS INC ELSEVIER SCIENCE
DOI: 10.1016/S1525-0016(03)00123-0
关键词
adeno-associated virus; retargeting; capsid mutants; combinatorial library; surface display; in vitro selection
Improving the efficiency and specificity of gene vectors is critical for the success of gene therapy. In an effort to generate viral mutants with controlled tropism we produced a library of adeno-associated virus (AAV) clones with randomly modified capsids and used it for the selection of receptor-targeting mutants. After several rounds of selection on different cell lines that were resistant to infection by wild-type (wt) AAV, infectious mutants were harvested at high titers. These mutants transduced target cells with an up to 100-fold increased efficiency, in a receptor-specific manner and without interacting with the primary receptor for wt AAV. The results demonstrate for the first time that a combinatorial approach based on a eukaryotic virus library allows one to generate efficient, receptor-specific targeting vectors with desired tropism.
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