Treatment of Conditions Caused By Calcium Abnormalities
AssigneeYale University
InventorsCraig M. Crews, Stephanie J. Quinn, Stefan Somlo, Dayne Okuhara
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Abstract
In certain aspects, the invention relates to use of PKD2 agonists, such as triptolide and triptolide derivatives, to regulate calcium release. In other aspects, the invention relates to use of PKD2 agonists to treat or aid in the treatment of any condition in which a calcium channel, such as the gene product of PKD 1 and/or PKD2, is mutated; calcium signaling is abnormal; or both, such as polycystic kidney disease.
Claims35
A method of treating or aiding in the treatment of polycystic kidney disease (PKD) in an individual in need thereof, comprising administering to the individual a therapeutically effective amount of a polycystin-2 (PKD2) agonist.
A method of treating or aiding in the treatment of a condition caused by abnormal calcium signaling, comprising administering to an individual in need thereof a therapeutically effective amount of a PKD2 agonist.
A method of treating a cystic disease in an individual in need thereof, comprising administering to the individual a therapeutically effective amount of a PKD2 agonist in an amount sufficient to slow or inhibit growth of cyst cells.
A method of slowing or inhibiting cyst formation, comprising contacting cyst cells with a PKD2 agonist in an amount sufficient to slow or inhibit growth of cyst cells.
A method of regulating calcium influx in a cell expressing polycystin-2, comprising contacting the cell with an effective amount of a PKD agonist.
A method of identifying a PKD2 agonist, comprising:
- (a) contacting a test agent to a cell expressing PKD2;
- (b) measuring PKD2-mediated calcium release in the cell;
- and
- (c) comparing the level of PKD2-mediated calcium release obtained in (b) with the level obtained in the absence of the test agent,
- wherein a greater level of PKD2-mediated calcium release in the presence of the test agent than in the absence of the test agent indicates that the test agent is a PKD2 agonist.
A method of identifying a therapeutic agent for slowing or inhibiting cyst formation, comprising:
- (a) contacting a test agent to a cell expressing PKD2;
- (b) measuring PKD2-mediated calcium release in the cell;
- and
- (c) comparing the level of PKD2-mediated calcium release obtained in (b) with the level obtained in the absence of the test agent,
- wherein a greater level of PKD2-mediated calcium release in the presence of the test agent than in the absence of the test agent indicates that the test agent is therapeutic agent for slowing or inhibiting cyst formation.
Use of a PKD2 agonist in the manufacture of medicament for the treatment of a cystic disease.
Use of a PKD2 agonist in the manufacture of medicament for the treatment of a condition caused by abnormal calcium signaling.
Dependencies are inferred from the claim text.