GENOMIC EDITING OF GENES INVOLVED IN SECRETASE-ASSOCIATED DISORDERS
First Claim
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1. A genetically modified animal comprising at least one edited chromosomal sequence encoding a protein associated with a secretase disorder.
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Abstract
The present invention provides genetically modified animals and cells comprising edited chromosomal sequences encoding proteins that are associated with a secretase disorder. In particular, the animals or cells are generated using a zinc finger nuclease-mediated editing process. Also provided are methods of using the genetically modified animals or cells disclosed herein to screen agents for toxicity and other effects.
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Citations
41 Claims
- 1. A genetically modified animal comprising at least one edited chromosomal sequence encoding a protein associated with a secretase disorder.
- 14. A non-human embryo, the embryo comprising at least one RNA molecule encoding a zinc finger nuclease that recognizes a chromosomal sequence encoding a protein associated with a secretase disorder, and, optionally, at least one donor polynucleotide comprising a sequence encoding an ortholog of the protein associated with a secretase disorder or an edited protein associated with a secretase disorder.
- 18. A genetically modified cell, the cell comprising at least one edited chromosomal sequence encoding a protein associated with a secretase disorder.
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26. A method for assessing the effect of an agent in a genetically modified animal, the method comprising administering the agent to the genetically modified animal comprising at least one edited chromosomal sequence encoding a protein associated with a secretase disorder, and comparing a parameter obtained from the genetically modified animal to the parameter obtained from a wild-type animal administered the same agent, wherein the 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) ability of the agent to modify an incidence or indication of a secretase disorder in the genetically modified animal. - View Dependent Claims (27, 28, 29, 30, 31, 32, 33)
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34. A method for assessing the therapeutic potential of an agent as a treatment for an secretase disorder, the method comprising administering the agent to a genetically modified animal, wherein the genetically modified animal comprises at least one edited chromosomal sequence encoding a protein associated with a secretase disorder, and comparing a selected parameter obtained from the genetically modified animal to the selected parameter obtained from a wild-type animal with no exposure to 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, 39, 40, 41)
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Specification