Gene Therapy
- Revolution of Medicine -
Diseases to Tackle!
Autism
One of the most physically disabling forms of Autism is the Rett syndrome, which happens to affect girls more. Besides the regressive form of autism, it also causes anxiety, inability to control speech, severe breathing, and even digestive problems. These are just few of the dire symptoms it has the potential to cause. Autism Speaks Senior Vice President for Scientific Affairs Andy Shih believes that gene therapy may be the best possible solution for this disease because the Rett gene happens to have many affects throughout the entire genome. So in order to tackle this disease, it may be best to actually go in and essentially correct the underlying genetic defect.
(citation 1.26)
Blindness
An incurable form of blindness, known as Choroideremia, affects 1 in 50,000 people. Those who have this condition tend to face worsening eye vision as they progress in age which as an end result, leaves them to be completely blind by their middle age. Although there is no known cure for this, researchers have found that perhaps they can insert a harmless virus, which would contain a healthy and functioning copy of the CHM gene. By doing so, this has the possibility of causing the photoreceptors in the eyes to no longer degenerate.
(citation 1.27)
Cancer
One type of cancer that Gene Therapy has potential to tackle is Leukemia. Leukemia is a "type of cancer that affects the blood and bone marrow" and is formed "when blood cells produced in the bone marrow grow out of control". (citation 1.28). In the United States alone, 1 person every 4 minutes is diagnosed with blood cancer. Gene Therapy can tackle this by "filtering patients' blood to remove millions of white blood cells called T-cells, altering them in the lab to contain a gene that targets cancer, and returning them to the patient in infusions over three days." (citation 1.29)
HIV
Human Immunodeficieny Virus (HIV) is affecting 33.4 million people today worldwide.(citation 1.30) It is a sexually transmitted disease, which attacks the most essential cells in your immune system: T-Cells and CD4 cells. Our bodies need these cells in order to fight off infections and diseases however HIV prevents this, and instead uses them to multiply itself and then destroy the cells. Gene Therapy can come in and change this because studies by the Sangamo Biosciences have just released a study in which this can be fixed by a technique called "gene editing". "The technique is designed to disrupt a gene, CCR5, used by HIV to infect T-cells, the white blood cells that fight viral infections. A patient's cells are removed and processed to alter the DNA that codes for the CCR5 receptor. The altered cells are multiplied and tested, then infused back into the patient." (citation 1.31)
*Please keep in mind that these are only the few out of MANY that Gene Therapy has the potential to tackle!
