Provided by Cognitive Sciences ePrint Archive
Published Quarterly
Mangalore, S outh India
ISSN 0972-5997
Volume 1; Issue 2; April-June 2002
Short Review
Gene T herapy - Potential, Pros, Cons And Ethics
Ananth N.
Department of Biochemistry, Center for Basic Sciences, Kasturba Medical College,
Mangalore - 575001, INDIA
Citation: Ananth N. Gene Therapy-Potential, Pros, Cons And Ethics. Online J Health Allied
Scs. 2002;2: 1
U R L : http://www.ojhas.org/issue2/2002-2-1 .htm
Open Acces s Archive: http: //cogprints.ecs.soton.ac.uk/view/subjects/OJHAS .html
Gene therapy offers an incredibly powerful tool for
preventing and curing disease and is based on aiding
the human body in fighting a disease or the expected
onset of a disease The genetic technology involved
with gene therapy is not based on altering the
human germ line as the misconception goes, a
practical truth that many fear misuse of. This type of
technology is more similar to medical treatments of
the past than most people realize; gene therapy
simply takes advantage of the human body's ability
to produce its own cure more quickly, safely and
effectively than can be done in a lab. Gene therapy
lends itself to numerous medical applications.
Bas ic gene ther apy appr oaches usually involve
adding something to a gene through a variety of
vectors . Most research and testing has been done
with the vector introduction of a sequence that codes
for a needed protein, either to counter a deficiency,
induce a strong immune response, or destroy tumor
cells. Examples of a needed protein introduced to
cure a disease include factor IX in hemophilia, and
the cystic fibrosis transmembrane conductance
regulator (CFTR).(1 ) Mutations in CFTR cause the
lethal genetic disease cystic fibrosis , but gene
therapy may be able to cure the disease by
introducing the wild-type CFT R into the individual.
Once the necessary sequence is incorporated into a
vector and introduced into the cells, the necessary
protein can be coded for. In this case, the human
body is directed into producing the required remedy
itself- gene therapy only gives it the plans it needs to
produce its own medicine.
Many genetic diseases are polygenic and do not
eas ily lend themselves to such corrective methods ad
hence the direct method mentioned above may not
be practical always. More effective, and most
popular , is another genetic ther apy approach that
primarily involves altering the immune system.
Although some individuals may be genetically
predis posed to contracting a certain disease due to
an immune deficiency, and therefore may need a
boosted immune response the most, most patients
could successfully combat their disease with an
increased immune response. Customized DNA
vaccines could encode for the cancer-specific
antigens of the patient. The vaccine would elicit an
enhanced immune response to eliminate the
unwanted cells.(1 ) Evidently here, gene therapy
s imply s tr engthens the body's own defense response
instead of attempting to cure the cancer with
methods that could harm the entire system. Even
genetically modified T lymphocytes expressing
s pecific r eceptor s designed to enhance their ability to
identify and destroy the cancer cells are being
developed.
T he immune response is not always helpful. Its
extreme sensitivity to foreign cells and their antigens
may be the first factor that has to be resolved,
es pecially when it is necessary to intr oduce foreign
cells into the body. When a transplant is performed,
not only is there a fear that the body's immune
system will attack the needed cells, but also that the
transplanted T-cells may attack some of the body's
vital organs such as the liver, gut and skin. To defeat
this disease called " graft-versus-host disease", a
1