Methods and Compositions for Treatment of Polycystic Kidney Disease
Provided herein are methods for the treatment of polycystic kidney disease, including autosomal dominant polycystic kidney disease, using modified oligonucleotides targeted to miR-17.
1 - 105 . (canceled)
106 . A method of treating polycystic kidney disease comprising administering to a human subject in need thereof a therapeutically effective amount of a modified oligonucleotide having the structure:
or a pharmaceutically acceptable salt thereof, wherein the polycystic kidney disease is autosomal dominant polycystic kidney disease.
107 . The method of claim 106 , wherein the pharmaceutically acceptable salt is a sodium salt.
108 . A method of treating polycystic kidney disease comprising administering to a human subject in need thereof a therapeutically effective amount of a modified oligonucleotide having the structure:
wherein the polycystic kidney disease is autosomal dominant polycystic kidney disease.
109 . The method of claim 106 , wherein the method comprises administering at least one additional therapy selected from an angiotensin II converting enzyme (ACE) inhibitor, an angiotensin II receptor blocker (ARB), a diuretic, a calcium channel blocker, a kinase inhibitor, an adrenergic receptor antagonist, a vasodilator, a benzodiazepine, a renin inhibitor, an aldosterone receptor antagonist, an endothelin receptor blocker, an mammalian target of rapamycin (mTOR) inhibitor, a hormone analogue, a vasopressin receptor 2 antagonist, an aldosterone receptor antagonist, a glucosylceramide synthase inhibitor, or an antihyperglycermic agent.
110 . The method of claim 106 , wherein the method comprises an additional therapy, wherein the additional therapy is dialysis.
111 . The method of claim 106 , wherein the method comprises an additional therapy, wherein the additional therapy is kidney transplant.
112 . The method of claim 107 , wherein the method comprises administering at least one additional therapy selected from an angiotensin II converting enzyme (ACE) inhibitor, an angiotensin II receptor blocker (ARB), a diuretic, a calcium channel blocker, a kinase inhibitor, an adrenergic receptor antagonist, a vasodilator, a benzodiazepine, a renin inhibitor, an aldosterone receptor antagonist, an endothelin receptor blocker, an mammalian target of rapamycin (mTOR) inhibitor, a hormone analogue, a vasopressin receptor 2 antagonist, an aldosterone receptor antagonist, a glucosylceramide synthase inhibitor, or an antihyperglycermic agent.
113 . The method of claim 107 , wherein the method comprises an additional therapy, wherein the additional therapy is dialysis.
114 . The method of claim 107 , wherein the method comprises an additional therapy, wherein the additional therapy is kidney transplant.
115 . The method of claim 108 , wherein the method comprises administering at least one additional therapy selected from an angiotensin II converting enzyme (ACE) inhibitor, an angiotensin II receptor blocker (ARB), a diuretic, a calcium channel blocker, a kinase inhibitor, an adrenergic receptor antagonist, a vasodilator, a benzodiazepine, a renin inhibitor, an aldosterone receptor antagonist, an endothelin receptor blocker, an mammalian target of rapamycin (mTOR) inhibitor, a hormone analogue, a vasopressin receptor 2 antagonist, an aldosterone receptor antagonist, a glucosylceramide synthase inhibitor, or an antihyperglycermic agent.
116 . The method of claim 108 , wherein the method comprises an additional therapy, wherein the additional therapy is dialysis.
117 . The method of claim 108 , wherein the method comprises an additional therapy, wherein the additional therapy is kidney transplant.