Grant Schiller's List: Genetics

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• What is gene therapy?
• This approach is most common.
• How does gene therapy work?
• Currently, the most common vector is a virus that has been genetically   altered to carry normal human DNA

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• Some of the different types of viruses used as gene therapy vectors:
• Current gene therapy is experimental and   has not proven very successful in clinical trials
• Before gene therapy can become   a permanent cure for any condition, the therapeutic DNA introduced into target   cells must remain functional and the cells containing the therapeutic DNA   must be long-lived and stable.
• the immune system is designed to attack the invader
• Conditions or disorders that arise from mutations   in a single gene are the best candidates for gene therapy.
• variations in many genes.

• What are some of the ethical considerations for using   gene therapy?

   

   --Some Questions to Consider...

   
   
  • What is normal and what is a disability or disorder, and who decides?
     
     
   
  • Are disabilities diseases? Do they need to be cured or prevented?
     
     
   
  • Does searching for a cure demean the lives of individuals presently affected   by disabilities?
     
     
   
  • Is somatic gene therapy (which is done in the adult cells of persons known   to have the disease) more or less ethical than germline gene therapy (which   is done in egg and sperm cells and prevents the trait from being passed on   to further generations)? In cases of somatic gene therapy, the procedure may   have to be repeated in future generations.
     
     
   
  • Preliminary attempts at gene therapy are exorbitantly expensive. Who will   have access to these therapies? Who will pay for their use?
   
   
• • what do You think about these issues?

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