Gene therapy is being used to combat a variety of health issues with positive outlooks. Bluebird Bio, a biotechnology company, may be able to significantly improve quality of life and health for those suffering from beta-thalassemia, a rare genetic blood disorder.
Beta-thalassamia is responsible for faulty links in the beta chain of hemoglobin, the transporter of oxygen throughout the human body. This genetic disorder requires regular blood transfusions for people who suffer from beta-thalassemia. The experimental gene therapy was administered to two patients who no longer needed blood transfusions after twelve days of treatment. A patient with a similar treatment was treated successfully, but it took longer for the effect to result to be positive, and the patient needed transfusions during the first year after the treatment started.
Lentivirus, a modified HIV virus is an experimental therapy Bluebird Bio uses as a replacement for the faulty beta globulin in gene stem cells. There are relatively few side effects according to a doctor involved in the research and treatment.
Scientists are claiming by altering how a liver gene works, they are fighting cholesterol with one injection, eliminating a need for daily pills in the reduction of the risk of heart attack. Harvard Stem Cell Institute and University of Pennsylvania scientists disrupted the activity of a gene, called PCSK9, in a test in mice, that regulates cholesterol. Levels of the lipid dropped by 35 to 40 percent in the process they administered, changing the genome permanently. The cholesterol health issue is a prime example of gene therapy combat yielding positive results.
The lead researcher, Kiran Musunnuru stated that was the same amount of cholesterol one would receive with a cholesterol drug. The exclamation point being the single injection, permanently changing the genome. Lipitor and Crestor are two pills targeting so-called bad cholesterol, designed to be taken on a daily basis. This new procedure may not replace pills for another 5 to 10 years. Therapy using genetics can be a form of combat when presented with health issues of this nature. It can sometimes be a battle that requires years of research to find the proper combinations for an outcome to be successful.
Working well in mice, there is not much of a leap to think this same therapy will work in humans as well. Stated Musunuru, who works in the Cambridge, Massachusetts school’s Department of Stem Cell and Regenerative Biology. Circulation Research, a journal of the American Heart Association, published the research on June 10, 2014.
The experiment using the genome-editing technique has been around for approximately 18 months. It is a two-part genome-engineering technique, it targets the DNA sequence where the gene sits first, and then creates a break in the system. Part two of this approach is to observe if the therapy is effective in human cells, replacing mice liver cells with human derived liver cells. Safety is a prime concern.
Another area of gene therapy with promise is using a virus as a delivery agent for successful treatment of malfunctioning genes in the brain. Most viruses are thought of as Trojan horses who deliver disease. This very trait makes them potential adversaries. A major problem in this area is coming up with the right Trojan horse for the job.
Several candidates are potential vectors (Trojan horse) in the search for gene therapy in brain diseases. Lentivirus, adenoviruss, and adeno-associated virus are the possible sources. Adenoviruses are also known as CAV-2 vectors, it was accidentally discovered they infect neurons, but not other types of brain cells.
Despite all the positive outlooks there is much more testing to be done, especially in the virus vector area. However, each step or phase of testing requires a positive result in all cases with few side effects. There is also the cost of testing and challenging research, in many cases a dead end is reached at the moment of brightest hope. Health issues using gene therapy appear as a form of combat when this occurs. The battle rages on.
By Andy Towle