The Food and Drug Administration (FDA) approved the First Gene Therapy For Blindness in the US on Tuesday-Luxturna of Spark Therapeutics, which treats a form of hereditary vision loss that can lead to blindness.
Researchers have been working for decades to change or replace defective genes with healthy genes to treat, cure or prevent disease or disease genes.
Now, this research is worth for gene therapy. Since August 2017, the US Food and Drug Administration. It has approved three gene products, the first of its kind.
Two of these are programmed to the cells themselves to attack a patient’s cancer tumor, and the newest approved product is targeted at a disease caused by mutations in a particular gene.
How Gene Therapy For Blindness works
Gene therapy for Blindness can be done both inside and outside the body. This graph easily shows how gene therapy works in the body.
Sometimes all or part of a gene that is defective or absent from birth, or a gene that may change or mutate in adulthood. Any of these variations can interfere with the way in which proteins are produced, which can contribute to health problems or diseases.
In gene therapy, researchers can do one of several things depending on the current problem. You can replace a gene that causes a medical problem with another add genes that help the body fight or treat diseases, or of genes that cause problems.
To insert new genes directly into the cells, the researchers use a vehicle called “vector”, which is genetically engineered to release the gene.
For example, viruses have a natural ability to deliver genetic material to cells and can therefore be used as vectors. However, before a virus can be used to carry therapeutic genes into human cells, it is modified to eliminate their ability to cause an infection disease.
Gene therapy can be used to change cells inside or outside the body. When the body is injected, a physician injects the vector that carries the gene directly into drugs that have broken cells.
In gene therapy, used to modify cells outside the body, blood, bone marrow or other tissue can be drawn from a patient and can separate specific cell types in the laboratory. The vector containing the desired gene is introduced into these cells. The cells are allowed to multiply in the laboratory and then reinjected into the patient where they continue to multiply and ultimately produce the desired effect.
Before a company can start a gene therapy for human use, the gene therapy product must be tested for safety and efficacy for FDA researchers to assess whether the risk of therapy is acceptable given the benefits.
Gene therapy promises to change medicine and create opportunities for patients living with serious and even incurable diseases. As researchers continue to make great progress in this treatment, the FDA is keen to accelerate development with an immediate review of innovative treatments that can save lives.
Sources: United States Food and Drug Administration