TARGETED CELL DEATH
First Claim
Patent Images
1. A recombinant nucleic acid molecule comprising:
- a nucleic acid sequence encoding a cell death mediator protein (CDMP), wherein said nucleic acid sequence is operably linked to a neural cell-specific regulatory element.
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Abstract
The present invention provides compositions and methods for studying neuropathy. The compositions and methods provided herein are particularly useful for screening agents of therapeutic and/or diagnostic potential.
53 Citations
70 Claims
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1. A recombinant nucleic acid molecule comprising:
- a nucleic acid sequence encoding a cell death mediator protein (CDMP), wherein said nucleic acid sequence is operably linked to a neural cell-specific regulatory element.
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2-11. -11. (canceled)
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12. A host cell comprising:
- a recombinant nucleic acid molecule comprising;
a nucleic acid sequence encoding a cell death mediator protein (CDMP), wherein said nucleic acid sequence is operably linked to a neural cell- specific regulatory element.
- a recombinant nucleic acid molecule comprising;
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13-29. -29. (canceled)
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30. A transgenic animal comprising:
- a nucleotide sequence encoding a cell death mediator protein (CDMP) operably linked to a cell type-specific expression regulatory element;
wherein said animal exhibits a greater degree of neuropathy relative to an animal without said nucleotide sequence. - View Dependent Claims (31, 32, 33, 34, 35, 36, 37, 38, 39, 40, 41, 42, 43, 44, 45, 46, 47, 48)
- a nucleotide sequence encoding a cell death mediator protein (CDMP) operably linked to a cell type-specific expression regulatory element;
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49. A method of screening for a biologically active agent that modulates a phenomenon associated with a demyelination or blood brain barrier disorder comprising:
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a) contacting a candidate agent with a cell comprising a nucleic acid encoding a cell death mediator protein (CDMP), wherein said nucleic acid is operably linked to a cell-type specific expression regulatory element; b) detecting an effect on said phenomenon; and
,c) selecting said agent as effective to modulate said phenomenon if the level of activity of said CDMP is modulated relative to a control cell.
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50. (canceled)
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51. (canceled)
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52. A method of screening for a biologically active agent that modulates a phenomenon associated with a demyelination or blood brain barrier disorder comprising:
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a) administering a candidate agent to a non-human transgenic animal, wherein said phenomenon occurs in said animal upon expression of a nucleic acid sequence encoding a cell death mediator protein (CDMP);
wherein said nucleic acid sequence expression is regulated by a cell-specific expression regulatory element;b) activating said CDMP to effect apoptosis in at least one cell in said animal, wherein said cell is associated with a demyelination blood brain barrier disorder; and
,c) detecting an effect of said agent upon said phenomenon.
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53-68. -68. (canceled)
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69. A method for compiling a profile data set for characterizing a phenomenon associated with multiple sclerosis(MS) or MS-associated condition related to multiple sclerosis comprising:
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a) providing a transgenic animal or cell comprising a nucleic acid encoding a cell death mediator protein (CDMP), wherein said nucleic acid is operably linked to a neuronal- or glial-specific expression regulatory element; b) activating said CDMP thereby inducing apoptosis; c) obtaining at least one surviving neuronal or glial cell following said activation; and d) profiling RNA transcripts and/or encoded products in said surviving glial or neuronal cell;
thereby compiling a profile data set characterizing a phenomenon associated with multiple sclerosis or MS-associated condition related to multiple sclerosis.
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70-72. -72. (canceled)
Specification