Genetic malfunctions are a common source of heartache for
many individuals and their families.
You probably personally know someone that is suffering right now. Medical research is consistently seeking
answers for us and for our loved ones who are currently afflicted. The Answer they have discovered-Gene Therapy.
The Answer for Genetic Diseases
Severe Combined Immune
Deficiency (ADA-SCID)
This genetic disease causes
children to be born with an ineffective immune system. Unless they can find a matched donor to
donate bone marrow for a transplant, they are stuck with living life in a “bubble”. That was until Gene therapy produced a
cure. Through gene therapy a gene
called ADA is administered into the patients bone marrow cells. This gene codes for the immune response
people with this disease are lacking.1
Hemophilia
Hemophilia can be life threatening. Individuals inflicted with this genetic malfunction are
unable to create blood clots. If
they are not careful they could have extreme blood loss either externally or
internally. Gene Therapy is
capable of giving these individuals the gene that causes normal blood clotting. This allows them to live a normal life
without added fear.1
Choroidermia
This disease causes progressive
loss of vision. In Childhood,
individuals will first loose night vision and eventually begin to narrow
sight. By Adulthood all of these
individuals are blind. This is
caused by gradual loss of cells in the back of the eye. Gene Therapy inserts genes and restores
eyesight. 2
These are just a few of many genetic disorders Gene Therapy has the potential to cure.
Gene Therapy the Answer for Cancer: A Success Story
Imagine
being the parent of beautiful vibrant 5-year-old girl. Suddenly you notice unusually large
bruises on your beloved child’s body.
Of course with worry you hurry her to the doctor, only to find that she
has leukemia. Years of medical
treatments will follow, along with much heartache and worry.
This
was the experience of the whitehead family.
Emily Whitehead had experienced three rounds of chemotherapy—enough to kill an
adult. Her tiny frame was
weakening and her future looked grim.
Miraculously, for her, she became part of University of Pennsylvania’s
gene therapy research. “Doctors took a sample of Whitehead's blood and isolated some of her T
cells (immunity cells). They then injected the sample with lentiviruses
(genetically modified HIV like viruses) that had been outfitted with the
appropriate genes. After a rocky start, which fortunately
responded to treatment, Whitehead quickly improved. Three weeks after
treatment, a quarter of the T cells in her bone marrow bore the genetic
correction. Her T cells began homing in on the cancer cells, which soon
vanished.”3 Through this
process this little girl’s body was able to kill her own cancer. Whitehead now has a full head of hair,
attends school and leads a normal life.
For Emily Whitehead, and 19 of 22 children who have received this treatment,
Gene Therapy was the answer.
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How Does Gene Therapy Work?
Gene Therapy is performed in a variety of different procedures, but the basic concept is pretty much the same. The
most common and developed method is by introducing a virus vector into affected
cells. These virus vectors are reconstructed, so that instead of causing a
disease in the host organism they incorporate their genetic material to fix the
host’s ineffective genes. Virus vectors come in different shapes and size with the choice depending
on the target cell and the nature of the genetic dysfunction. However, the basic concept of each is the same: to fix the base problem within genetic material.
The Retrovirus
The retrovirus can only target dividing cells because within it is only RNA. After entering the cell the virus releases the RNA into the nucleus. Then as the cell is replicating in preparation for division, the genetic code from the virus is incorporated directly into the cell’s DNA. While the cell and its daughter cells continue to divide, more cells with the incorporated virus DNA are created to perform the missing function. The virus that saved Emily Whiteheads life falls under this species of virus.
The Adenovirus
Click Here: More Info on How Virus Vectors Work
Sources:
1. http://drugdiscovery.com2. http://ghr.nlm.nih.gov/condition/choroideremia
3. http://web.b.ebscohost.com
Picture Sources:
1. http://biol1020-1.blogspot.com/2010_05_01_archive.html
3. http://www.techtimes.com/
4. http://emilywhitehead.com/
5. http://www.odec.ca/projects/2007/wood7a2/gt1.htm
