GENOME EDITING OF IMMUNODEFICIENCY GENES IN ANIMALS
First Claim
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1. A genetically modified animal comprising at least one edited chromosomal sequence encoding an immunodeficiency protein.
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Abstract
The present invention provides genetically modified animals and cells comprising edited chromosomal sequences encoding immunodeficiency proteins. In particular, the animals or cells are generated using a zinc finger nuclease-mediated editing process. Also provided are methods of assessing the effects of agents in genetically modified animals and cells comprising edited chromosomal sequences encoding immunodeficiency proteins.
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38 Claims
- 1. A genetically modified animal comprising at least one edited chromosomal sequence encoding an immunodeficiency protein.
- 14. A non-human embryo, the embryo comprising at least one RNA molecule encoding a zinc finger nuclease that recognizes a chromosomal sequence encoding an immunodeficiency protein, and, optionally, at least one donor polynucleotide comprising a sequence encoding an immunodeficiency protein.
- 18. A genetically modified cell, the cell comprising at least one edited chromosomal sequence encoding an immunodeficiency protein.
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29. A method for assessing the effect of an agent in an animal, the method comprising contacting a genetically modified animal comprising at least one edited chromosomal sequence encoding an immunodeficiency protein with an agent, and comparing results of a selected parameter to results obtained from contacting a wild-type animal with the same agent, wherein the selected parameter is chosen from:
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a) rate of elimination of the agent or its metabolite(s); b) circulatory levels of the agent or its metabolite(s); c) bioavailability of the agent or its metabolite(s); d) rate of metabolism of the agent or its metabolite(s); e) rate of clearance of the agent or its metabolite(s); f) toxicity of the agent or its metabolite(s); and g) efficacy of the agent or its metabolite(s). - View Dependent Claims (30, 31, 32, 33)
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34. A method for assessing the therapeutic potential of an agent in an animal, the method comprising contacting a genetically modified animal comprising at least one edited chromosomal sequence encoding an immunodeficiency protein with an agent and comparing results of a selected parameter to results obtained from a wild-type animal with no contact with the same agent, wherein the selected parameter is chosen from:
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a) spontaneous behaviors; b) performance during behavioral testing; c) physiological anomalies; d) abnormalities in tissues or cells; e) biochemical function; and f) molecular structures. - View Dependent Claims (35, 36, 37, 38)
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Specification