GENOMIC EDITING OF GENES INVOLVED IN ALZHEIMER'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 AD.
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Abstract
The present invention provides genetically modified animals and cells comprising edited chromosomal sequences encoding proteins associated with AD. 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 AD development and methods of assessing the effects of agents in genetically modified animals and cells comprising edited chromosomal sequences encoding proteins associated with AD.
122 Citations
50 Claims
- 1. A genetically modified animal comprising at least one edited chromosomal sequence encoding a protein associated with AD.
- 15. 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 AD, and, optionally, at least one donor polynucleotide comprising a sequence encoding a protein associated with AD.
- 19. A genetically modified cell, the cell comprising at least one edited chromosomal sequence encoding a protein associated with AD.
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32. 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 NO;
7, 8, 9, 10, 11, and 12; andb) a cleavage domain. - View Dependent Claims (33, 34, 35)
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36. 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 NO:
- 7, 8, 9, 10, 11, and 12.
- View Dependent Claims (37)
- 38. A method for assessing the effect of a genetically modified protein associated with AD on the progression or symptoms of AD or an AD-related disorder 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 AD, and measuring an AD-related phenotype.
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45. A method for assessing the effect of an agent on progression or symptoms of AD, the method comprising:
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a) contacting a first genetically modified animal comprising at least one edited chromosomal sequence encoding a protein associated with AD with an agent; b) measuring an AD-related phenotype in the first genetically modified animal, and c) comparing results of the AD-related phenotype in (b) to results obtained from a second genetically modified animal comprising the same edited chromosomal sequence as the first genetically modified animal, wherein the second genetically modified animal is not contacted with the agent. - View Dependent Claims (46, 47, 48, 49, 50)
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Specification