When does Gene Therapy Work?

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WHAT IS GENE THERAPY?

HISTORY OF GENE THERAPY

WHEN DOES GENE THERAPY WORK?

WHEN DOES GENE THERAPY NOT WORK?

WHAT ARE THE ETHICS OF GENE THERAPY?

OTHER AREAS GENE THERAPY CAN BE APPLIED

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          The obvious question is when is it possible to use gene therapy to treat a genetic disease?   This is a tough question to approach since gene therapy is still a relatively new idea and hasn't been researched to it's full potential yet.  However, there are several circumstances where gene therapy should theoretically work.  To some extent scientists hope that one day all genetic diseases would be treatable by gene therapy.  A more realistic hope is that all genetic diseases caused by a single gene can be treated by gene therapy.  Why should this have any effect on gene therapy?  To answer this question you have to understand the complexity of many of the bodily functions that most people see as a simple task.  Take the metabolism of a simple sugar for instance.  While to most people without a science background this seems like it would probably be a simple process controlled by a few genes.  The reality is that basic carbohydrate metabolism is a very complicated process requiring far more than a few proteins and enzymes.  On the other hand there are many genetic diseases that should be treatable with gene therapy since they have been linked to to one or two genes.  Examples of such diseases include Alzheimer's Disease, Huntington's disease, and Parkinson's Disease.  By the end of 1993 gene therapy had already been approved for severe combined immune deficiency, familial hypercholesterolemia, cystic fibrosis, and Gaucher's disease. 
          Below is a list from the National Reference Center for Bioethics Literature at Georgetown University.
 

 




 

     Criteria for Diseases to be Treated by Gene Therapy
                                  (III, Nichols1988, p. 18)

   1) the disease is an incurable, life-threatening disease

   2) organ, tissue and cell types affected by the disease have been identified

   3) the normal counterpart of the defective gene has been isolated and
       cloned

   4) the normal gene can be introduced into a substantial subfraction of the
       cells from the affected tissue; or that introduction of the gene into the
       available target tissue, such as bone marrow, will somehow alter the
       disease process in the tissue affected by the disease

   5) the gene can be expressed adequately (it will direct the production of
       enough normal protein to make a difference)

   6) techniques are available to verify the safety of the procedure.

 

 

 

This page was created as part of the ISSI 470 class at Monmouth College.  All ideas are the property of the author.