Gene therapy is a remarkable technique that researchers are developing to treat several types of diseases. Although the use of this medical therapy for humans is still in its infancy, gene therapy (GT) has the potential to completely change the prognoses for millions of ill people throughout the world. Of all of the remarkable medical developments of the past decade, GT is clearly one of the most impressive. Simply, GT involves strategically introducing DNA into the patient under treatment. Typically, the new DNA is used because it contains a well-functioning gene chosen to fix the effects of a harmful genetic mutation.
Researchers are still in the early stages of developing GT to treat human patients. Until now, the technique has not proven very effective for curing humans. However, recent breakthroughs have brought GT closer to widespread use in medicine. Thanks to these brilliant medical breakthroughs, doctors might soon use GT to treat muscular dystrophy (MD), cystic fibrosis and a variety of genetic disorders.
Gene augmentation therapy is perhaps the most simple form of GT. This is the form of GT that uses implanted DNA to correct genetic mutations. For example, a particular gene might not produce a protein needed by the human body. To correct the mutation, doctors introduce DNA that contains a more effective version of the malfunctioning gene. This therapy has the potential to treat medical disorders that are classified as reversible. Cystic fibrosis is disease that may soon respond to treatment of this type. It is a truism that people must take responsibility for their own health outcomes. Nevertheless, the public sector has a definite obligation to help improve the state of public health.
Though GT has enormous potential for the future, some obstacles remain before this therapy is ready for widespread adoption. Still, a recent news development demonstrated that GT has a lot of staying power as a medical discipline. In early September, 2017, the FDA approved a trial of a new GT technique for treating leukemia. This represents the very first time that any national government has given approval for this type of human trial. This type of GT made headlines worldwide when it was used to save the life of a sick British child named Layla.
Called CAR-T, this therapy involves resetting the patient's immune cells to make them more adept at targeting cancer cells. A sample of the patient's own T-cells is sent to a pharmaceutical laboratory located in New Jersey. At the laboratory, technicians use an inert virus to insert a gene into the patient's T-cells. This added gene will give the patient's immune system far more ability to hone in on the out-of-control cells that cause cancer. If people want to live in societies that take proper care of the ill, they should encourage public officials to support GT research. Without doubt, this avenue of research is ripe for development. With proper funding and support, it seems sure that GT will evolve into a powerful, easily accessible treatment that will ease the lot of millions.
If CAR-T is fully tested and approved, which seems fairly likely, this will represent a major milestone in the history of American health care. CAR-T is designed to treat the type of leukemia involved in the vast majority of child leukemia cases. Nevertheless, this treatment will provide hope for patients of all ages and their loved ones. The projected price for one treatment will be under $500, which is fairly reasonable for leukemia treatments in this day and age. This treatment is priced so that even patients with fairly humble means will have the opportunity to experience cutting-edge care.
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