‘IN BODY’ GENE EDITING

About: 

• The studies involve men with Hunter or Hurler Syndrome, diseases caused by a missing gene that makes an enzyme to break down certain sugar compounds. Without it, sugars build up and damage organs, often killing people in their teens. 
  
  
• Working: 
  • In 2017, Brian Madeux of Arizona became the first person to try it. Through an IV, he received many copies of a corrective gene and an editing tool called zinc finger nucleases to insert it into his DNA. 
    
    
  • Joseph Muenzer of the University of North Carolina at Chapel Hill, helped test the treatment, with the help of the therapy's maker, California-based Sangamo Therapeutics. 
    
    
  
  
  
• Findings: 
  • Results on him and seven other Hunter patients, plus three with Hurler Syndrome, suggest the treatment is safe, which was the main goal of these early experiments. 
    
    
  • Preliminary results suggest that two men with a rare disorder now have a corrective gene at very low levels, which may not be enough to make the therapy a success. 
    
    
  • Still, it’s a scientific milestone, a first step toward one day doctoring DNA to treat many diseases caused by faulty genes. 
    
    
  
  
  
• Significance: 
  • In body Gene editing is intended as a more precise way to do gene therapy, to disable a bad gene or supply a good one that’s missing. 
    
    
  • Trying it in adults to treat diseases is not controversial and the DNA changes do not pass to future generations, unlike the recent case of a Chinese scientist who claims to have edited twin girls’ genes when they were embryos.