GENOMIC EDITING OF GENES INVOLVED WITH PARKINSON'S DISEASE
First Claim
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1. A genetically modified animal comprising at least one edited chromosomal sequence encoding a protein associated with Parkinson'"'"'s disease.
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Abstract
The present invention provides genetically modified animals and cells comprising edited chromosomal sequences encoding proteins associated with Parkinson'"'"'s disease. 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 PD development and screen agents for assessing their effect on progression or symptoms of PD.
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Citations
41 Claims
- 1. A genetically modified animal comprising at least one edited chromosomal sequence encoding a protein associated with Parkinson'"'"'s disease.
- 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 Parkinson'"'"'s disease, and, optionally, at least one donor polynucleotide comprising a sequence encoding a protein associated with Parkinson'"'"'s disease.
- 18. A genetically modified cell, the cell comprising at least one edited chromosomal sequence encoding a protein associated with Parkinson'"'"'s disease.
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27. A zinc finger nuclease, the zinc finger nuclease comprising:
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a) a zinc finger DNA binding domain that binds a sequence having at least about 80% sequence identity with a sequence chosen from SEQ ID NOs;
3, 4, 5, 6, 7, 8, 9, 10, 11, and 12; andb) a cleavage domain. - View Dependent Claims (28, 29, 30)
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31. A nucleic acid sequence bound by a zinc finger nuclease, the nucleic acid sequence having at least about 80% sequence identity with a sequence chosen from SEQ ID NOs:
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3, 4, 5, 6, 7, 8, 9, 10, 11, and 12. - View Dependent Claims (32)
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33. A method for assessing the effect of a genetically modified protein associated with PD on the progression of PD in an animal, the method comprising comparing a wild type animal to a genetically modified animal comprising at least one edited chromosomal sequence encoding a protein associated with Parkinson'"'"'s disease, and measuring a selected parameter, wherein the selected parameter is chosen from:
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a) amyloidogenesis; b) protein aggregation; c) response to dopamine; d) neurodegeneration; e) mitochondrial dysfunction; f) coordination; g) balance; h) gait; i) motor impairment; j) tremors and twitches; k) rigidity; l) hypokinesia; and m) cognitive impairment. - View Dependent Claims (34, 35, 36)
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37. A method for assessing the effect of an agent on progression or symptoms of PD, the method comprising contacting a first genetically modified animal comprising at least one edited chromosomal sequence encoding a protein associated with Parkinson'"'"'s disease with the agent, and comparing the results of a selected parameter to results obtained from a second genetically modified animal not contacted with the agent, wherein the first and second genetically modified animals each comprise chromosomal sequences that have been edited exactly the same, the selected parameter being chosen from:
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a) amyloidogenesis; b) protein aggregation; c) response to dopamine; d) neurodegeneration; e) mitochondrial dysfunction; f) coordination; g) balance; h) gait; i) motor impairment; j) tremors and twitches; k) rigidity; l) hypokinesia; and m) cognitive impairment. - View Dependent Claims (38, 39, 40, 41)
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Specification