GENOMIC EDITING OF GENES INVOLVED IN AUTISM SPECTRUM DISORDERS
First Claim
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1. A genetically modified animal comprising at least one edited chromosomal sequence encoding a protein associated with ASD.
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Abstract
The present invention provides genetically modified animals and cells comprising edited chromosomal sequences encoding proteins associated with ASD. 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 study ASD development and screen agents for assessing their effect on progression or symptoms of an ASD.
123 Citations
41 Claims
- 1. A genetically modified animal comprising at least one edited chromosomal sequence encoding a protein associated with ASD.
- 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 ASD, and, optionally, at least one donor polynucleotide comprising a sequence encoding an ortholog of the protein associated with ASD or an edited protein associated with ASD.
- 18. A genetically modified cell, the cell comprising at least one edited chromosomal sequence encoding a protein associated with ASD.
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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 ASD, 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 an ASD in the genetically modified animal. - View Dependent Claims (27, 28, 29, 30, 31, 32)
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34. A method for assessing the therapeutic potential of an agent as a treatment for an ASD, 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 ASD, 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 (33, 35, 36, 37, 38, 39, 40, 41)
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Specification