âA Special Report from Victor Marchione, MD
Gene therapy is a subject that has divided the medical community. Some feel that altering the genetic code is akin to playing God. Others see gene therapy as a way to wipe out genetic diseases from the human gene pool permanently.
What is gene therapy? Simply put, gene therapy is the substitution of defective genes in a cell with the correct ones. Genes, of course, are the basic heredity material in our bodies. So gene therapy involves supplying a healthy, working gene to cells that either do not work properly or do not work at all. The aim of gene therapy is to repair a genetic disorder or acquired disease.
There are two categories of gene therapy. The first involves the alteration of germ cells, that is, sperm or eggs. This is the form of gene therapy that many people react to on an ethical basis, because it permanently alters the genetic material of the whole organism, as well as future generations. This type of therapy is commonly called “germ line gene therapy.” The second type of gene therapy is called “somatic cell gene therapy.” It is somewhat like getting an organ transplant. Specific cells are targeted and replaced with healthy genetic material. This type of gene therapy does not alter the genetic material of the treated person’s offspring.
When thinking about genetic therapy, it is important to keep in mind the aforementioned distinctions. Gene therapy can be a powerful tool that could potentially help in the treatment of diseases that have, up until this point, had no treatment at all.
For example, researchers at the University of Oklahoma Health Sciences Center have just made a discovery that will have huge implications for people suffering from retinitis pigmentosa. They have found a way to use a radical new type of gene therapy to prevent the blindness associated with this condition.
The research team discovered a way to deliver gene therapies directly to the light-sensitive cells affected by retinitis pigmentosa. With the help of nano-particle technology, scientists created a microscopic capsule capable of carrying genetic therapies to their destination inside the cells of the retina. The delivery of this tiny capsule is now being tested in a variety of gene therapies. The scientists hope the new therapy method also has the potential to treat a wide range of diseases from bladder cancer to diabetes.
For the study, researchers were astounded when they witnessed the capsules carrying therapies to the designated location in the eye within 15 minutes of delivery and spreading the genetic repair message quickly to nearby cells.
This discovery is already being used to develop new treatments for macular degeneration, the researchers say. The medical community is now describing this breakthrough treatment as “breathtaking” and “very exciting.” The use of this therapy could help give independence and drastically improved quality of life to hundreds of thousands of people suffering from degenerative eye conditions.