Gene Therapy: Bane or Blessing ? dna75.jpg (12900 bytes)

Introduction The Task | ResourcesThe Process  |  Learning AdviceConclusion

Introduction- What is gene therapy ?  it is an appproach to treating disease through the manipulation of an individual's genes.  Currently gene therapy is being developed to treat a variety of genetic diseases and disorders as well as vacular disease, cancer, immune deficiencies, and neruodegernerative diseases.  Of the 100,000 genes which comprise the human genome many have been linked to the malfunction of a single gene.  Cystic fibrosis has been linked to mutations in a gene called the Cystic Fibrosis Transmembrane Regulator( CFTR) which is located on chromosome number seven. Cystic fibrosis is an autosomal recessively inherited respiratory disease resulting in the accumlation of mucus on the surface of the lungs due to the distortion of a membrane protein for the transport of the chloride ion.   Patients treated by conventional methods are living longer more productive lives with advances in medicine, but still succomb to infections.  Cystic fibrosis is one of the genetic diseases for which gene therapy has been developed.

The Task

You are a member of the committee which will meet to decide whether gene therapy will be initiated at your hospital,Suburbia East.  In order to make the decision it will be necessary to investigate the following.


Resources on gene therapy


Web Quest Links

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Gene Therapy. Bane or Blessing ?

Honors Genetics. Cherokee High School

Instructor- P. Sidelsky

Gene Therapy- General References -   Official policy on gene therapy    Applications of gene therapy   Magazine containing articles on different applications of

gene therapy. Sample Issue – Transplantation and gene therapy reference. The reason for gene therapy in transplantation techniques is explained. - Human Molecular Gene Therapy . Abstracts and articles from the magazine - National Organization for rare Disorders. Rare Genetic Disease dataBase  – A complete reference for gene therapy

Universities and Medical Center- Gene Therapy – Baylor University gene therapy site. Pediatric and adult gene therapy linked to stem cells. - Gene Therapy and Cancer links  – Gene Therapy for vascular genetic diseases  - Institute for Human Gene Therapy at the University of Pennsylvania. This has many links on the Jesse Gelsinger Case. Sept. 1999  - Gene Therapy in Transplantation On becoming a gene therapy patient - Human Gene Therapy Research Institute

Commercial Gene Therapy Products - Commercial site for the development of retroviral vectors - Suicide gene kits for cancer cells. - Biomedical site - Gene Therapy Clinical Trials - A diagram of a model vector in Adeno-associated virus.

Bioethics – Bioethics and Gene Therapy

Additional References


In Search of an Answer


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  1.  Divide the class in groups of four students.

  2.  Assign each group to investigate the implementation of gene therapy in the treatment of disease from a different perspective.

a.  the molecular research scientist

b.  the medical team

c.  the legal consultants

d.  the bioethical group

e.  the clinical geneticists and genetic counselors

3. Each group should prepare a presentation consisting of the following

a. Position paper

b.  Audiovisual aids including an overhead ( Power Point presentation may be substituted)

c.  Bibliographic references.  Web sites used for reference.  Any new sites discovered and described will result in additional credit.

d. All members of the group should contribute to the preparation of the final product.

Gene Investigation Tips

Information is added to the web sites in the reference section daily. Use a variety of web sites and sources to reach your opinion on the issue of gene therapy.  You should include factual references for the justification of your opinion.

Grading Rubric

Category 1 2 3 4 5
Use of references
Visual display
Group effort

Clearly stated

Total for group work


1 - Weak. Needs additional effort. All categories deficient

2 - Below average. Some categories need additional work.

3 - Average. Meets basic requirements

4 -Shows creativity and initiative

5 -Superior in content, presentation, and preparation

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Conclusion - Each group should form a consensus opionion based on its research.  The consensus should be clearly stated with support for its position in a well written paragraph.


This page written by Patricia L. Sidelsky
Last updated date. May 23, 2000

This page was adapted from Bernie Dodge 's WebQuest_Template1.html by Tom March