Map kinase pathway targets for the treatment of marfan syndrome

Abstract

The instant disclosure provides methods and compositions for the diagnosis, treatment and prevention of Marfan syndrome and related diseases, disorders and conditions. The disclosure further provides pharmaceutical compositions and kits for the diagnosis, treatment and prevention of Marfan syndrome and related diseases, disorders and conditions.

1 . A method for treating a subject having or at risk of developing Marfan Syndrome or a Marfan-associated condition comprising:
 administering to the subject an effective amount of an agent that modulates the activity of MAP kinase pathway signaling; thereby treating the subject.   
     
     
         2 . The method of  claim 1 , wherein the agent that modulates the activity of MAP kinase pathway signaling is an inhibitor of the MAP kinase pathway. 
     
     
         3 . The method of  claim 1 , wherein the agent that modulates the activity of MAP kinase pathway signaling is an inhibitor of MMP17, MAP2K6 or MAP3K4, or of a gene product thereof. 
     
     
         4 - 5 . (canceled) 
     
     
         6 . The method of  claim 1 , wherein the agent that modulates the activity of MAP kinase pathway signaling is a small molecule. 
     
     
         7 . The method of  claim 1 , wherein the agent that modulates the activity of MAP kinase pathway signaling comprise Batimastat, GI 254023X, GM 6001, TMI 1, WAY 170523, WX-554 or combinations thereof. 
     
     
         8 . (canceled) 
     
     
         9 . The method of  claim 1 , wherein the Marfan syndrome-associated disease or disorder is a clinical condition associated with Marfan syndrome. 
     
     
         10 . The method of  claim 9 , wherein the disease or disorder is an aneurysm, an aortic aneurysm, or emphysema. 
     
     
         11 . The method of  claim 9 , wherein the disease or disorder is an aneurysm. 
     
     
         12 - 39 . (canceled) 
     
     
         40 . The method of  claim 1 , further comprising an inhibitor of transforming growth factor β (TGFβ). 
     
     
         41 . (canceled) 
     
     
         42 . The method of  claim 3 , wherein the agent that modulates the activity of MAP kinase pathway signaling is a small molecule.