Gene Therapy: The Answer

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. 

 Watch Her Story on YouTube

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 

This virus, unlike the retrovirus, contains full DNA and does not fuse with the cell’s original DNA.  Instead, when it is injected into the nucleus it immediately begins giving the cell the needed instructions to make a missing protein or other necessary and previously nonfunctioning missing element. 

Click Here: More Info on How Virus Vectors Work

Sources:

1.  http://drugdiscovery.com
2.  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

2.  http://drugdiscovery.com

3.  http://www.techtimes.com/

4.  http://emilywhitehead.com/

5.  http://www.odec.ca/projects/2007/wood7a2/gt1.htm