Brief History of Gene Therapy

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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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          "On September 14, 1990 researchers at the U.S. National Institutes of Health performed the first (approved) gene therapy procedure on four-year old Ashanti DeSilva. Born with a rare genetic disease called severe combined immune deficiency (SCID), she lacked a healthy immune system, and was vulnerable to every passing germ. Children with this illness usually develop overwhelming infections and rarely survive to adulthood; a common childhood illness like chickenpox is life-threatening. Ashanti led a cloistered existence--avoiding contact with people outside her family, remaining in the sterile environment of her home, and battling frequent illnesses with massive amounts of antibiotics. (http://www.georgetown.edu/research/nrcbl/scopenotes/sn24.html)"

          Since 1990 there have been several clinical trials for different types of gene therapies.  However, little progress has been made since those early trials in 1990.  Gene therapy suffered a major setback in 1999 after 18 year old Jesse Gelsinger died just 4 days after receiving gene therapy treatment.  Jesse was part of a trial for Transcarboxylase deficiency.  He died from an acute immune response to the retrovirus used to deliver the gene.  Since 1999 there have been several other cases of patients developing a rare form of leukemia that is believed to be linked to the gene therapy.  In January of 2003 the FDA placed a temporary ban on all retroviral gene therapy because of the link to leukemia.   

 

 

 

 

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