Gene Therapy - Isaac Chan
Updated: 6/5/2020
Gene Therapy - Isaac Chan
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  • Gene Therapy
  • If you'll follow me.
  • Yes! So what did you have in mind?
  • While it is not perfect, gene therapy would be your best option. With gene therapy we will add the correct version of the gene causing DMD.
  • You said you have tried all other treatments for Duchenne muscular dystrophy (DMD)?
  • Some things you should know:
  • However, the risk is that your son may have an immune response, meaning the treatment won't work, in addition to feeling weaker.
  • The good thing about gene therapy is that it can treat genetic disorders that are otherwise difficult to treat or untreatable, like DMD.
  • If it saves him, I think we should do it!
  • Ok. We will first get a copy of the correct gene then use a viral vector, a virus that will carry the gene, and administer it to your son through an injection.
  • After this first dose we will see how the treatment works. After that your son will have to come back every while as this is not permanent.
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