The Future of Medicine: Gene Therapy and Treatments

DNA-1Imagine a world where all known diseases were curable, where health problems could be treated in a non-invasive manner, and life could be extended significantly? Thanks to ongoing research into the human genome, and treatments arising out of it, that day may be coming soon. That’s the idea behind gene therapy and pharmacoperones – two treatment procedures that may make disease obsolete in the near future.

The first comes to us from the Utah School of Medicine, where researcher Amit Patel recently developed a non-invasive, naked DNA approach to deal with treating heart problems. His process was recently tested o Ernie Lively, an actor suffering from heart damage, who made a full recovered afterwards without ever having to go under the knife.

gene_therapyIn short, Patel’s method relies on a catheter, which he used to access the main cardiac vein (or coronary sinus), where a balloon is inflated to halt the flow of blood and isolate the area. A high dose of naked DNA, which codes for a protein called SDF-1, is then delivered. SDF-1, which stands for stromal cell-derived factor, is a potent attractant both for stem cells circulating in the bloodstream, and for those developing in the bone marrow.

Stromal cells, which manufacture SDF-1, are the creative force which knit together our fibrous connective tissues. The problem is they do not make enough of this SDF-1 under normal conditions, nor do specifically deliver it in just the right places for repair of a mature heart. By introducing a dose of these cells directly into the heart, Patel was able to give Lively what his heart needed, where it needed it.

gene_therapy1Compared to other gene therapies, the introduction of SDF-1 into cells was done without the assistance of a virus. These “viral vector” method have had trouble in the past due to the fact that after the virus helps target specific cells for treatment, the remnant viral components can draw unwanted attention from the immune system, leading to complications.

But of course, there is still much to be learned about the SDF-1 treatment and others like it before it can be considered a viable replacement for things like open-heart surgery. For one, the yield – the number or percentage of cells that take up the DNA – remains unknown. Neither are the precise mechanisms of uptake and integration within the cell known here.

Fortunately, a great deal of research is being done, particularly by neuroscientists who are looking to control brain cells through the use of raw DNA as well. Given time, additional research, and several clinical trials, a refined version of this process could be the cure for heart-related diseases, Alzheimer’s, and other disorders that are currently thought to be incurable, or require surgery.

pharmacoperones-protein-foldingAnother breakthrough treatment that is expected to revolutionize medicine comes in the form of pharmacoperones (aka. “protein chaperones”). a new field of drugs that have the ability to enter cells and fix misfolded proteins. These kind of mutations usually result in proteins becoming inactive; but in some cases, can lead to toxic functionality or even diseases.

Basically, proteins adopt their functional 3-D structure by folding linear chains of amino acids, and gene mutation can cause this folding process to go awry, resulting in “misfolding”. Up until recently, scientists believed these proteins were simply non-functional. But thanks to ongoing research, it is now known their inactivity is due to the cell’s quality control system misrouting them within the cell.

protein1Although this process has been observed under a microscope in recent years, a team led by Doctor P. Michael Conn while at Oregon Health & Science University (OHSU) was the first to demonstrate it in a living laboratory animal. The team was able to cure mice of a disease that makes the males unable to father offspring, and believe the technique will also work on human beings.

The team says neurodegenerative diseases, such as Alzheimer’s, Parkinson’s and Huntington’s, as well as certain types of diabetes, inherited cataracts and cystic fibrosis are just a few of the diseases that could potentially be cured using the new approach. Now working at the Texas Tech University Health Sciences Center (TTUHSC), Conn and his team are looking to conduct human trials.

DNA-molecule2One of the hallmarks of the coming age of science, technology and medicine is the idea that people will be living in post-mortality age, where all diseases and conditions are curable and life can be extended almost indefinitely. Might still sound like science fiction, but all of this research is indicative of the burgeoning trend where things that were once thought to be “treatable but not curable” is a thing of the past.

It’s an exciting time to be living in, almost as exciting as the world our children will be inhabiting – assuming things go according to plan. And in the meantime, check out this video of the SDF-1 gene therapy in action, courtesy of the University of Utah School of Medicine:


Sources: extremetech.com, gizmag.com

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