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Adeno-associated viral vector-based methods and compositions for introducing an expression cassette into a cell
| Details |
Inventors: Nakai, Hiroyuki; Kay, Mark A.;
Assignee: The Board of Trustees of the Leland Stanford Junior University (Palo Alto, CA)
Primary Examiner: Ketter; James
Assistant Examiner: Marvich; Maria
Attorney, Agent or Firm: Field; Bret E. Bozicevic, Field & Francis, LLP
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. |
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DETAILED DESCRIPTION What is claimed is: 1.
A method for introducing a functional expression cassette into a cell, said method comprising: (a) providing a population of first and second adeno-associated viral particles, wherein said first adeno-associated viral particle comprises a first portion of an expression cassette and said second adeno-associated viral particle comprises a second portion of an expression cassette; and (b) contacting said cell with said first and second adeno-associated viral particles under conditions sufficient for a hetero-concatemer to be produced inside said cell, wherein said hetero-concatemer comprises a functional expression cassette made up of said first and second portions separated by an intron comprising an inverted terminal repeat (ITR) sequence, wherein said contacting occurs in vitro; to introduce a functional expression cassette into said cell.
2.
The method according to claim 1, wherein said functional expression cassette comprises DNA capable of being transcribed into an mRNA molecule.
3.
The method according to claim 1, wherein said functional expression cassette is longer than about 4.
7 kb.
4.
The method according to claim 1, wherein said population comprises only two distinct adeno-associated viral vectors.
5.
A kit for use in introducing a functional expression cassette into a cell, said kit comprising: first and second plasmid vectors for producing a population of at least two distinct adeno-associated viral particles, wherein each distinct member of said population of at least two distinct adeno-associated viral particles comprises a different portion of said single an expression cassette that produce a hetero-concatemer comprising a single expression cassette; and instructions for practicing the method according to claim 1.
6.
The kit according to claim 5, wherein said first plasmid vector comprises an insertion site flanked by adeno-associated virus inverted terminal repeat (ITR) sequences.
7.
The kit according to claim 5, wherein said second plasmid vector comprises Rep and Cap coding sequences
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Gene Therapy 
