The Future of Gene Therapy
The past few years have been eventful for Gene Therapy. Finally, after decades of waiting and unwanted failures, clear results and successes are now being reported from various Gene Therapy from their clinical trials, research studies, regulatory approvals, and effective new methods.
To put it simply, Gene therapy is a superb genetic engineering method. In Gene therapy, abnormal genes from the human body are specifically targeted by foreign genes so that during the process they will be repaired, modified or replaced with healthy genes.
In this article, we will highlight the exciting innovation, and wonderful improvements, successful studies and a promise of a bright future in Gene Therapy field:
Gene Therapy Cures Blood Disorder Thalassemia
A student at Arizona State University participated in a gene therapy study to treat her genetic blood disorder condition called as Beta Thalessemia. The leading scientists on the study aimed to reduce or fully eliminate the need for quarterly blood transfusion for those who are suffering from Beta Thalessemia blood disorder. During the study, Her body was transplanted with her own genetically modified genes that were cultivated and modified to treat her Thalassemia disorder. Post the gene therapy study, the student was monitored for some time and she reported that the study went swimmingly well and that she has been transfusion-free ever since.
Say No More to Inherited Vision Loss
US Food and Drug Administration (FDA) approved the first gene therapy in the US called Luxturna that is aimed at treating patients with a vision loss caused by a rare genetic disorder. Moreover, the new gene therapy is suited to treat young children and adults alike. The company behind Luxturna is Spark Therapeutics. The company priced the one-time gene therapy treatment at a whopping $850,000. As of now, further data is required to check its effectiveness however the company remains hopeful that with the approval of FDA they can get more clients willing to undergo the process stating that the price is well justified.
First Gene-Editing Therapy
Brian Madeux is suffering from Hunter syndrome, a genetic disorder that is affecting his liver condition, so he decided to become the first human experience for gene editing. Prior to the experiment, his life was only expected to last after a few years according to the doctor’s diagnosis. So far, he is proving them wrong and wishes to overcome the disease. After 5 months since the test, Brian and the group of doctors working on his case has not noticed any major setbacks and the test is performing as expected. Brian is helpful that by volunteering to be a part of this test in the hope of finding a treatment for future generations suffering from the same genetic disorder. Read more about the history of gene therapy
In France, gene therapy researchers successfully treated a boy’s sick-cell disease. This disease is a blood disorder that degrades hemoglobin, an integral part of red blood cells, function to flow freely and delivery of oxygen in the body.
Scientists removed defective and mutating genes from the bone marrow and performed gene in the lab by introducing copies of a gene to prevent his red blood cells from changing into the sickled blood. When the treated cells were infused back into his body, they began to make normal blood cells. After 2 years, the patient, now a teenager, lives a normal life without needing to have blood transfusions, other intrusive procedures and, so far, there were no side effects noticed.
Skin Disease Epidermolysis Bullosa Corrected
A young boy had been borne with a genetic disorder that affects his skin. His skin disorder is called Epidermolysis Bullosa which, if left untreated, can result in a fatal condition since the skin blister profusely even with minor trauma or friction. The leading advancements in gene-editing and correcting had fixed the problem by replacing the abnormal genes and skin with grafted and genetically modified cells. The doctors consider this study as one of the vital cases that will decide where the future of gene therapy lies. After 1 month, the boy’s body had adjusted well with the treatment and 80% of his body is now covered in strong and think epidermis as well as not developing any blisters or mucous membrane in the grafted areas.
Duchenne muscular dystrophy (DMD) Clinical Trial Approved
US Food and Drug Administration (FDA) has approved several studies involving gene therapy. One, in particular, is a clinical trial for Duchenne muscular dystrophy (DMD) that aims to cure one of the most common and fatal genetic disorders affecting countless children all over the world. DMD is devastating and, without gene therapy, a rather incurable and muscle degenerating diseases. The clinical trials have released data that shows a very promising result. The academes involved with the trial have all their hands on deck and to ensure that this will be a pivotal study trial where they can usher a new paradigm that will transform outcomes for patients afflicted with the same genetic disorder so that they can live normal lives.
It sure is an exciting time for Gene Therapy and the healthcare system in general. With a lot of positive news and study results success, the future looks bright and may signal the revolution of a new era in how diseases are cured or, better yet, prevented. In the future, it may even be possible to treat a fetus for the genetic disease even before getting born. Scientists remain hopeful that the key lies in the human genome composition to unlock the potential to cure diseases and result in wonderful opportunities for the advancement of the field (Read more about gene therapy benefits). Without a doubt, Gene therapy a one of the highly debated, and hottest area of medical research today. It is only a matter of time before it is made available to everyone at a much cheaper price.
Do you approve of the various innovations Gene Therapy showcased in the past years? If so, please like and share this article. Are there other Gene Therapy method you wish were improved? Please leave a comment below. Thanks!