Adeno-associated viral vector-based methods and co

专利名称：Adeno-associated viral vector-based

methods and compositions for introducingan expression cassette into a cell

发明人：Hiroyuki Nakai,Mark A. Kay申请号：US09797858申请日：20010301

公开号：US20020058341A1公开日：20020516

摘要：Methods and compositions are provided for introducing an expression cassetteinto a cell. In the subject methods, a population of at least two distinct adeno-associatedviral particles is provided, where each distinct type of viral particle in the populationcomprises 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 underconditions sufficient to produce a hetero-concatemer in the cell, where the hetero-concatemer includes a functional expression cassette having an intron that includes anITR sequence. Also provided by the subject invention are vector preparations forpracticing the subject methods as well as kits for use in producing the vectors employedin the subject methods. The subject methods find use in a variety of different genetransfer applications, including both in vivo and in vitro gene transfer applications, and areparticularly suited for use in the transfer of long genes.

申请人：NAKAI HIROYUKI,KAY MARK A.

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