Interview about Gene Therapy

With Dr. Hemal Amin, M.D. from Kaiser Permanente in Roseville, CA

 Question 1: Can you please briefly explain what Gene Therapy is?

• Dr. Hemal Amin: "Gene therapy is an experimental technique that uses genes to correct defective genes responsible for disease development or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery" (citation 4.1).

 Question 2: What are some risks involved with this procedure?

• Dr.Hemal Amin: 
• " Immune response - Anytime a foreign object is introduced into human tissues, the immune system is designed to attack the invader. The risk of stimulating the immune system in a way that reduces gene therapy effectiveness is always a potential risk. Furthermore, the immune system's enhanced response to invaders it has seen before makes it difficult for gene therapy to be repeated in patients" (citation 4.2).
• Problems with viral vectors - Viruses, while the carrier of choice in most gene therapy studies, present a variety of potential problems to the patient --toxicity, immune and inflammatory responses, and gene control and targeting issues. In addition, there is always the fear that the viral vector, once inside the patient, may recover its ability to cause disease" (citation 4.2).

Question 3: Is gene therapy right for everyone? Or is it only limited to certain people? If so, why or how?

• Dr. Hemal Amin: "Gene therapy is currently only being tested for the treatment of diseases that have no other cures" (citation 4.1).  

Question 4: Is gene therapy currently being used? Why or why not?

• Dr. Hemal Amin: "Gene therapy is a promising treatment option for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections). It is still in experimental stage.The technique remains risky and is still under study to make sure that it will be safe and effective" (citation 4.3) 

Question 5: What are some ethical and religious issues that are causing a hindrance to the advancement of Gene Therapy?

• Dr.Hemal Amin: These are the unanswered questions regarding gene therapy:
  • "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?" (citation 4.4)