GENOME EDITING OF GENES ASSOCIATED WITH SCHIZOPHRENIA IN ANIMALS
First Claim
Patent Images
1. A genetically modified animal comprising at least one edited chromosomal sequence encoding a protein associated with schizophrenia.
2 Assignments
0 Petitions
Accused Products
Abstract
The present invention provides genetically modified animals and cells comprising edited chromosomal sequences associated with schizophrenia. In particular, the animals or cells are generated using a zinc finger nuclease-mediated editing process. The invention also provides zinc finger nucleases that target chromosomal sequence associated with schizophrenia and the nucleic acids encoding said zinc finger nucleases. Also provided are methods of assessing the effects of agents in genetically modified animals and cells comprising edited chromosomal sequences associated with schizophrenia.
-
Citations
49 Claims
- 1. A genetically modified animal comprising at least one edited chromosomal sequence encoding a protein associated with schizophrenia.
- 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 schizophrenia, and, optionally, at least one donor polynucleotide comprising a sequence encoding an ortholog of the protein associated with schizophrenia or an edited protein associated with schizophrenia.
- 18. A genetically modified cell, the cell comprising at least one edited chromosomal sequence encoding a protein associated with schizophrenia.
-
29. A zinc finger nuclease, the zinc finger nuclease comprising:
-
a) a zinc finger DNA binding domain that binds a sequence having at least about 80% sequence identity to a sequence chosen from SEQ ID NOs;
4, 5, 6, and 7; andb) a cleavage domain. - View Dependent Claims (30, 31, 32)
-
-
33. A nucleic acid sequence recognized by a zinc finger nuclease, the nucleic acid sequence having at least about 80% sequence identity with a sequence chosen from SEQ ID NOs:
- 4, 5, 6 and 7.
-
34. 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 schizophrenia, 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:
-
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 a schizophrenia-related disorder in the genetically modified animal. - View Dependent Claims (35, 36, 37, 38, 39, 40, 41)
-
-
42. A method for assessing the therapeutic potential of an agent as a treatment for a schizophrenia-related disorder, 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 schizophrenia, 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:
-
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 (43, 44, 45, 46, 47, 48, 49)
-
Specification