Gene therapy and stem cells unite
        
            By James Gallagher, 
                BBC News
             | 10. 13. 2011
        
                    
                                    
                    
                                                                                                                                    
                                                                            
                              
    
  
  
    
  
          
  
      
    
            
Two of the holy grails of medicine - stem cell technology and precision gene therapy - have been united for the first time in humans, say scientists.
It means patients with a genetic disease could, one day, be treated with their own cells.
A study in Nature corrected a mutation in stem cells made from a patient with a liver disease.
Researchers said this was a "critical step" towards devising treatments, but safety tests were still needed.
At the moment, stem cells created from a patient with a genetic illness cannot be used to cure the disease as those cells would also contain the corrupted genetic code.
Scientists, at the Wellcome Trust Sanger Institute and the University of Cambridge, were working on cirrhotic liver disease.
It is caused by a change to a single pair of letters, out of the six billion which make up the genetic code.
As a result, a protein which protects the body from damage, antitrypsin, cannot escape from the liver where it is made.
The illness is one of the most common genetic diseases, affecting...
 
       
 
  
 
    
    
  
   
                        
                                                                                
                 
                                                    
                            
                                  
    
  
  
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