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=Gene Therapy: The Future of Medicine =

Introduction/Overview
Gene therapy has been an evolving science for many years. Scientists involved in the research of gene therapy have transformed the future of medicine. The idea of infusing the human body with a cell that would reverse a disease inherited at birth was once a long shot. Today scientists have experimented with many case studies and, even though the procedure is not yet perfected, researchers and scientists are much closer to an outcome people can live with.

The concepts of gene therapy were made during the 1960s and early 1970s while the development of genetically marked cells lines and the clarification of mechanisms of cell transformation was in progress. With the arrival of recombinant DNA techniques, cloned genes became available and were used to demonstrate that foreign genes could indeed correct genetic defects and disease phenotypes in mammalian cells in vitro. Efficient retroviral vectors and other gene transfer methods have permitted convincing results of efficient phenotype correction in vitro and in vivo, now making gene therapy a broadly accepted approach to therapy and justifying clinically applied studies with human patients.

The Food and Drug Administration (FDA) has not yet approved any human gene therapy product for sale. Current gene therapy is experimental and has not proven very successful in clinical trials. Little progress has been made since the first gene therapy clinical trial began in 1990. In 1999, gene therapy suffered a major setback after the death of 18-year-old Jesse Gelsinger.

Gene therapy has changed the course of medicine for the better. Not only is gene therapy improving throughout the years, but scientists are getting amazing results. Over 4,200 diseases have been identified as resulting directly from abnormal genes. Many people around the world live with diseases and gene therapy will be their cure.



History/Chronology of Events
In the early 1970's, scientists came up with the idea of "gene surgery" for treating inherited diseases that are caused by damaged genes. Their idea was to take out the disease-causing gene and surgically put in a gene that functions properly. Throughout history society has been questioning the ability of gene therapy. Many fatal outcome have come into play since society has made their opinion, but gene therapy is much more than society has made it to be.

Researchers have come up with a surgical way to implant undamaged genes into the human body to correct damaged cells. Scientists have been discovering new ways to infuse the body with these cells ever since earlier scientists proposed the idea of gene therapy. Today there are many ways to inject the body with undamaged cells, however, this surgery has not yet been perfected. Because this science has not been perfected, many humans have died during the clinical trial. These outcomes are why society is skeptical about the idea of gene therapy.

Legislation, Facts and/or Statistical Data
The first human gene therapy trial began in 1990 and treated two patients with Severe Combined Immune iorder (SCID) caused by ADA deficiency. In a 1996 survey, 68% of Americans approved of germline and somatic gene therapy. In the same survey, 83% of physicians supported use of germline gene therapy to eradicate diseases. Worldwide there are 396 gene therapy protocols involving 3278 patients (Sept. 1999). 75% of all gene therapy trials take place in the United States. 63% of all approved gene therapy protocols are for trials for treating cancer. 40% of Americans think it would be okay to use gene therapy to make their children more attractive or intelligent. Primary limitation in gene therapy: efficient gene delivery to body.

Case Study
Jess Gelsinger, a 18-year-old boy was the first fatal victim of gene therapy. Jesse was participating in a gene therapy trial for ornithine transcarboxylase deficiency (OTCD). He died from multiple organ failures 4 days after starting the treatment. His death is believed to have been triggered by a severe immune response to the adenovirus carrier. During the informed consent process, Jesse and his father, Paul, were told that potentially the most serious side effect would be hepatitis and the need for a liver transplant but that the possibility of either of these reactions was low. Paul said they were not told about two previous trial participants who has experienced serious side effects, nor about two monkeys that had died in animal trials using the same vector virus. This information was not in the consent document. These facts were brought to light and now more strick evaluation and informed consent rules have to be taken in order for an individual to be entered into a clinical trial.

Biographical Sketches of atleast 2 Key Individuals related to topic
Dr. Theodore Friedman Dr. Teodore Friedman and another scientists wrote an article on gene therapy called "Gene Therapy for Human Disease?" alerting the public to the technical and ethical issues on the horizon. Since then he has performed many gene therapy clinical trials. [|Lab] Jesse Gelsinger Jesse Gelsinger was a 17-year-old boy who suffered from OTC. He was entered into a clinical trial where he died. His death changed the course of gene therapy because of the missing steps doctors took before he was entered into the trial. [|In Memory of Jesse Gelsinger]

Glossary of Terms
Gene Therapy: Gene therapy is the transfer of abnormal genes to normal genes into the body Stomatic Therapy: Exchanging of abnormal cells anywhere in the body Germ Line Therapy: Exchanging of abnormal cells in the reproductive organs Recombinant DNA: Form of artificial DNA that is created by combining two or more sequences that would not normally occur together through the process of gene splicing.

Recomended Research Databases
Opposing Viewpoints in Context Science in Context Health and Wellness Resource Center Global Issues in Context

Print Sources:
Finn, Jeffrey, and Eliot L Marshall. Medical Ethics. Ed. Dale C Garell, M.D. and Solomon H Snyder, M.D. Vol. 1. 1990. New York: Chelsea House Publishers, 1990. Print. Panno, Joseph, Ph.D. Gene Therapy: Treating Disease by Repairing Genes. Illus. Richard Garratt and Joseph Panno. New York: Facts On File, Inc., 2005. Print.

Recomended Websites:
[|Gene Therapy News] [|American Society of Gene and Cell Therapy]