Use of Lyso-GB1 as Druggable Target

Abstract

The present invention is related to the in vitro use of lyso-Gb1 as a draggable target in the development of a drug, and to antagonist of lyso-Gb1 for use in the treatment and/or prevention of a disease, wherein the disease is Gaucher disease or Parkinson's disease

1 .- 15 . (canceled) 
     
     
         16 . A method of using lyso-Gb1 as a druggable target in the development of a drug antagonizing lyso-Gb1 by physical interaction for the treatment and/or prevention of Parkinson's disease, wherein the method comprises:
 determining in vitro whether a drug candidate binds to lyso-Gb1 present outside of a cell, tissue or organ,   determining in vivo whether the drug candidate is antagonizing the action of lyso-Gb1 resulting in ameliorating at least one symptom of the disease; wherein the drug candidate is a drug for the treatment and/or prevention of Parkinson's disease if the at least one symptom is ameliorated,   and wherein lyso-Gb1 is of formula (I)   
       
         
           
           
               
               
           
         
       
     
     
         17 . The method of  claim 16  further comprising determining the binding affinity of the drug candidate to lyso-Gb1. 
     
     
         18 . The method of  claim 16 , wherein the drug candidate is identified in a screening process from a library of compounds, wherein the screening process comprises providing a library of compounds and identifying from the library of compounds one or more drug candidates capable of binding to lyso-Gb1. 
     
     
         19 . The method of  claim 16 , wherein Parkinson's disease is caused by a mutation of glucocerebrosidase. 
     
     
         20 . The method of  claim 16 , wherein Parkinson's disease is selected from the group consisting of a mild form of Gaucher disease, type 1 Gaucher disease and non-neuronopathic Gaucher disease. 
     
     
         21 . The method of  claim 16 , wherein the at least one symptom is a central nervous symptom. 
     
     
         22 . The method of  claim 16 , wherein the at least one symptom is selected from the group consisting of ataxia, dementia, ocular apraxia, and parkinsonism.