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.
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