发明名称 Adeno-associated viral vector-based methods and compositions for introducing an expression cassette into a cell
摘要 Methods and compositions are provided for introducing an expression cassette into a cell. In the subject methods, a population of at least two distinct adeno-associated viral particles is provided, where each distinct type of viral particle in the population comprises a different portion of the expression cassette to be introduced into the cell. The target cell is contacted with population of adeno-associated viral vectors under conditions sufficient to produce a hetero-concatemer in the cell, where the hetero-concatemer includes a functional expression cassette having an intron that includes an ITR sequence. Also provided by the subject invention are vector preparations for practicing the subject methods as well as kits for use in producing the vectors employed in the subject methods. The subject methods find use in a variety of different gene transfer applications, including both in vivo and in vitro gene transfer applications, and are particularly suited for use in the transfer of long genes.
申请公布号 US7060497(B2) 申请公布日期 2006.06.13
申请号 US20010797858 申请日期 2001.03.01
申请人 THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITY 发明人 NAKAI HIROYUKI;KAY MARK A.
分类号 C12N15/63;A01N63/00;C07H21/02;C12N15/00;C12N15/864;C12P21/06 主分类号 C12N15/63
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