GENOME EDITING OF GENES ASSOCIATED WITH TRINUCLEOTIDE REPEAT EXPANSION DISORDERS IN ANIMALS
First Claim
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1. A genetically modified animal comprising at least one edited chromosomal sequence encoding a protein associated with a trinucleotide repeat expansion 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 trinucleotide repeat expansion disorders. 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
36 Claims
- 1. A genetically modified animal comprising at least one edited chromosomal sequence encoding a protein associated with a trinucleotide repeat expansion 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 trinucleotide repeat expansion disorder, and, optionally, at least one donor polynucleotide comprising a sequence encoding a protein associated with a trinucleotide repeat expansion disorder.
- 18. A genetically modified cell, the cell comprising at least one edited chromosomal sequence encoding a protein associated with a trinucleotide repeat expansion disorder.
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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 a protein associated with a trinucleotide repeat expansion disorder with the 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)
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33. 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 a protein associated with a trinucleotide repeat expansion disorder 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 (34, 35, 36)
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Specification