CANINE GENOME EDITING WITH ZINC FINGER NUCLEASES
First Claim
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1. A genetically modified canine comprising at least one edited chromosomal sequence encoding a canine or human disease-related protein.
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Abstract
The present invention provides a genetically modified canine or cell comprising at least one edited chromosomal sequence. In particular, the chromosomal sequence is edited using a zinc finger nuclease-mediated editing process. The disclosure also provides zinc finger nucleases that target specific chromosomal sequences in the canine genome.
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Citations
32 Claims
- 1. A genetically modified canine comprising at least one edited chromosomal sequence encoding a canine or human disease-related protein.
- 9. A canine embryo comprising at least one RNA molecule encoding a zinc finger nuclease that recognizes a chromosomal sequence encoding a canine or human disease-related protein, and, optionally, at least one donor polynucleotide comprising a sequence encoding a canine or human disease-related protein.
- 12. A genetically modified canine cell, the cell comprising at least one edited chromosomal sequence encoding a canine or human disease-related protein.
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19. A method for assessing the effect of an agent in a canine, the method comprising contacting a genetically modified canine comprising at least one edited chromosomal sequence encoding a canine or human disease-related protein, and comparing results of a selected parameter to results obtained from contacting a wild-type canine 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 (20, 21, 22, 23)
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24. A method for assessing the therapeutic potential of an agent in a canine, the method comprising contacting a genetically modified canine comprising at least one edited chromosomal sequence encoding a canine or human disease-related protein, and comparing results of a selected parameter to results obtained from a wild-type canine with 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 (25, 26, 27, 28)
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Specification