Gene Therapy: Bane or Blessing ?

Introduction |  The Task | Resources |  The Process  |  Learning Advice |  Conclusion

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.

• Investigate the benefits of gene therapy to the treatment of genetic disorders,vascular disease, immune deficiencies, neurodegenerative diseases, and cancer ( Clinical Genetics Team )

• Investigate the regulations and protocols for gene therapy.  Official guidelines for the operation of a clinic. ( Legal advisors )

• Investigate potential problems from prior cases of gene therapy. ( Medical staff )

• Invetigate new research on vectors and administration of gene therapy from a commercial standpoint ( Research molecular biologist)

• Evaulate the ethics of the protocol( risk versus reward )

Resources on gene therapy

Web Quest Links

Gene Therapy. Bane or Blessing ?

Honors Genetics. Cherokee High School

Instructor- P. Sidelsky

AEPSidelsk@aol.com

Gene Therapy- General References

http://www.mdanderson.org/depts/genetherapy/    Applications of gene therapy

http://www.stockton-press.co.uk/gt –    Magazine containing articles on different applications of

gene therapy. Sample Issue

http://www-ermm.cbcu.cam.ac.uk/99000691h.htm – Transplantation and gene therapy reference. The reason for gene therapy in transplantation techniques is explained.

http://www3.oup.co.uk/hmg/Volume_09/Issue_09/ - Human Molecular Gene Therapy . Abstracts and articles from the magazine

http://www.rarediseases.org/ - National Organization for rare Disorders. Rare Genetic Disease dataBase

http://www.nih.gov/index.html  – A complete reference for gene therapy

Universities and Medical Center- Gene Therapy

http://www.bcm.tmc.edu/bmt/content_01a.html – Baylor University gene therapy site. Pediatric and adult gene therapy linked to stem cells.

http://www.bcm.tmc.edu/genetherapy/links_00.html - Gene Therapy and Cancer links

http://www.bcm.tmc.edu/genetherapy/links_00.html  – Gene Therapy for vascular genetic diseases

http://www.med.upenn.edu/ihgt/  - Institute for Human Gene Therapy at the University of Pennsylvania. This has many links on the Jesse Gelsinger Case. Sept. 1999

http://www-ermm.cbcu.cam.ac.uk/99000691h.htm  - Gene Therapy in Transplantation

http://www.mdanderson.org/patient/ On becoming a gene therapy patient

http://www.hgtri.org/main.htm - Human Gene Therapy Research Institute

http://nt.onnet.co.kr:8888/genemed/right.html

Commercial Gene Therapy Products

http://www.natx.com/ - Commercial site for the development of retroviral vectors

http://www.invivogen.com/ - Suicide gene kits for cancer cells.

http://www.invivogen.com/ - Biomedical site

http://www.wiley.co.uk/genetherapy/clinical/ - Gene Therapy Clinical Trials

http://www.plasmed.com/ - A diagram of a model vector in Adeno-associated virus.

Bioethics

http://www.ornl.gov/hgmis/publicat/hgn/v10n1/13white.html – Bioethics and Gene Therapy

Additional References

• http://bioinformatics.weizmann.ac.il/cards/   Gene Cards can be used to research a variety of genetic disease and disorder by category or by information on the gene

• http://www.ncbi.nlm.nih.gov/omim/ On- line Mendelian Inheritance in Man provides detailed information on genetic diseases and predisposition  to genetic disorders

• http://www.ornl.gov/hgmis/resource/education.html, Human Genome Project information including recent research news
• http://ehrweb.aaas.org/ehr/books/   Your Genes Your Choices

In Search of an Answer

  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

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

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