Gene therapy has historically been used to treat disorders with in-depth knowledge caused by a single genetic mutation. Thanks to the introduction of new generation technologies, the potential of gene therapy is expanding tAo treat diseases that were previously untreatable.
Evolution of Gene Therapy
One of the major success stories of the twenty-first century has been gene therapy.
However, it has not been the same in the past. The field's journey to this point has been long and mostly difficult, with both tragedy and triumph along the way.
Initially, genetic disorders were thought to be untreatable and permanently carved into the genomes of individuals unfortunate enough to be born with them. But due to the constant technological advancement and research activities, gene therapy now has the potential to treat various genetic mutation-causing diseases with its ability to insert a new copy and replace faulty genes.
Gene Therapy is Finding New Roads in the Medical Sector
Gene therapy can help researchers treat a variety of conditions that fall under the general heading of epilepsy, instead of only focusing on a particular kind of disorder brought on by a genetic mutation. Following are some of the domains transformed by gene therapy.
– Gene therapy can be used for the treatment of seizures by directly injecting it into the area causing an uncontrolled electrical disturbance in the brain. Furthermore, by using DNA sequences
known as promoters, gene therapy can be restricted to specific neurons within that area.
– Genetic conditions such as blindness can be caused due to the mutation of any gene out of over 200 and resulting in progressive vision loss in children. With advanced gene therapies such as optogenetics,
lost photoreceptor function can be transferred to the retinal cells, which are responsible for relaying visual information to the brain. This might give patients the ability to navigate in an unknown environment with a certain level of autonomy.
The Future of Gene Therapy
The news surrounding gene therapy has been largely favorable over the past few years, with treatment after treatment obtaining regulatory approvals, successful clinical trials, and garnering significant funds to begin development. With more than 1,000 clinical trials presently underway, the long-awaited gene therapy revolution might finally be here.