COMPOSITIONS AND METHODS FOR THE TREATMENT OR PREVENTION OF MITOCHONDRIAL DISEASES
First Claim
Patent Images
1. A method of reducing the number of defective mitochondria in a cell, the method comprising(a) identifying a cell as having an increased number of defective mitochondria;
- (b) contacting the cell with an agent that increases Pink1 or Parkin expression or biological activity in the cell, thereby reducing the number of defective mitochondria in the cell.
1 Assignment
0 Petitions
Accused Products
Abstract
The present invention features compositions and methods for the treatment or prevention of diseases associated with a mitochondrial defect.
-
Citations
56 Claims
-
1. A method of reducing the number of defective mitochondria in a cell, the method comprising
(a) identifying a cell as having an increased number of defective mitochondria; (b) contacting the cell with an agent that increases Pink1 or Parkin expression or biological activity in the cell, thereby reducing the number of defective mitochondria in the cell. - View Dependent Claims (3, 6, 8, 10, 19, 20)
-
2. (canceled)
-
4-5. -5. (canceled)
-
7. (canceled)
-
9. (canceled)
-
11-13. -13. (canceled)
- 14. A method of treating or preventing a mitochondrial disease in a subject, the method comprising identifying the subject as having an increased number of defective mitochondria and administering to the subject an effective amount of an agent that increases Pink1 or Parkin expression or biological activity in a cell, thereby treating the disease.
-
18. (canceled)
-
21-35. -35. (canceled)
-
36. A method for identifying a compound useful for the treatment of a mitochondrial disease, the method comprising:
-
(a) contacting a cell with a compound and an agent that disrupts mitochondrial function; and (b) identifying a reduction in the number of defective mitochondria in the cell relative to a control cell not contacted with the candidate compound, wherein a compound that reduces the number of defective mitochondria in the cell is identified as useful for the treatment of a mitochondrial disease;
ora method for identifying a compound useful for the treatment of a subject having a mitochondrial disease, the method comprising; (a) contacting a cell derived from the subject with a compound and an agent that disrupts mitochondrial function; and (b) identifying a reduction in the number of defective mitochondria in the cell relative to a control cell not contacted with the candidate compound, wherein a compound that reduces the number of defective mitochondria in the cell is identified as useful for the treatment of the subject having mitochondrial disease;
ora method for identifying a compound useful for the treatment of a mitochondrial disease, the method comprising; (a) contacting a cell with a compound and an agent that disrupts mitochondrial function; and (b) identifying an increase of PINK1 or Parkin associated with mitochondria in the cell relative to a control cell not contacted with the candidate compound, wherein a compound that increases PINK1 or Parkin association with mitochondria in the cell is identified as useful for the treatment of a mitochondrial disease;
ora method for identifying a compound useful for the treatment of a mitochondrial disease, the method comprising; (a) contacting a cell comprising a mutation in mitochondrial DNA with a compound; and (b) identifying a reduction in the number of defective mitochondria in the cell relative to a control cell not contacted with the candidate compound, wherein a compound that reduces the number of defective mitochondria in the cell is identified as useful for the treatment of a mitochondrial disease;
ora method for identifying a compound useful for the treatment of a mitochondrial disease, the method comprising; (a) contacting a cell with a compound; and (b) identifying an increase of PINK1 or Parkin associated with mitochondria in the cell relative to a control cell not contacted with the candidate compound, wherein a compound that increases PINK1 or Parkin association with mitochondria in the cell is identified as useful for the treatment of a mitochondrial disease;
ora method for identifying a compound useful for the treatment of a subject having a mitochondrial disease, the method comprising; (a) contacting a cell derived from the subject with a compound; and (b) identifying a reduction in the number of defective mitochondria in the cell relative to a control cell not contacted with the candidate compound, wherein a compound that reduces the number of defective mitochondria in the cell is identified as useful for the treatment of said mitochondrial disease in the subject;
ora method for identifying a compound useful for the treatment of Parkinson'"'"'s disease, the method comprising; (a) contacting a dopaminergic cell with a candidate compound and an agent that disrupts mitochondrial function; and (b) identifying a reduction in the number of defective mitochondria in the cell relative to a control cell not contacted with the candidate compound, wherein a compound that reduces the number of defective mitochondria in the cell is identified as useful for the treatment of a Parkinson'"'"'s disease;
ora method for identifying a compound useful for the treatment of Parkinson'"'"'s disease, the method comprising; (a) contacting a cell comprising a mutation in Pink1 or Parkin with a candidate compound; and (b) identifying a reduction in the number of defective mitochondria in the cell relative to a control cell not contacted with the candidate compound, wherein a compound that reduces the number of defective mitochondria in the cell is identified as useful for the treatment of a Parkinson'"'"'s disease. - View Dependent Claims (46)
-
-
37-45. -45. (canceled)
-
47-50. -50. (canceled)
- 51. A method for ameliorating Parkinson'"'"'s disease in a subject, the method comprising administering to the subject an agent that reduces the biological activity or expression of PARL.
-
54. (canceled)
Specification