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Gene therapy is the addition of new genes to a patient's cells to replace missing or malfunctioning genes. Researchers typically do this using a virus to carry the genetic cargo into cells, because that's what viruses evolved to do with their own genetic material.

The treatment, which was first tested in humans in 1990, can be performed inside or outside of the body. When it's done inside the body, doctors may inject the virus carrying the gene in question directly into the part of the body that has defective cells. This is useful when only certain populations of cells need to be "fixed." For example, researchers are using it to try to treat Parkinson's disease, because only part of the brain must be targeted. This approach is also being used to treat eye diseases and hemophilia, an inherited disease that leads to a high risk for excess bleeding, even from minor cuts.

Early in-the-body gene therapies used a virus called adenovirus-the virus behind the common cold-but the agent can cause an immune response from the body, putting a patient at risk of further illness. Today, researchers use a virus called adeno-associated virus, which is not known to cause any disease in humans. In nature, this agent needs to hitch a ride with an adenovirus, because it lacks the genes required to spread itself on its own. To produce an adeno-associated virus that can carry a therapeutic gene and live on its own, researchers add innocuous DNA from adenovirus during preparation.

In-the-body gene therapies often take advantage of the natural tendency of viruses to infect certain organs. Adeno-associated virus, for example, goes straight for the liver when it is injected into the bloodstream. Because blood-clotting factors can be added to the blood in the liver, this virus is used in gene therapies to treat hemophilia.

In out-of-the-body gene therapy, researchers take blood or bone marrow from a patient and separate out immature cells. They then add a gene to those cells and inject them into the bloodstream of the patient; the cells travel to the bone marrow, mature and multiply rapidly, eventually replacing all of the defective cells. Doctors are working on the ability to do out-of-the-body gene therapy to replace all of a patient's bone marrow or the entire blood system, as would be useful in sickle-cell anemia-in which red blood cells are shaped like crescents, causing them to block the flow of blood.

Out-of-the-body gene therapy has already been used to treat severe combined immunodeficiency-also referred to as SCID or boy-in-the-bubble syndrome-where patients are unable to fight infection and die in childhood. In this type of gene therapy, scientists use retroviruses, of which HIV is an example. These agents are extremely good at inserting their genes into the DNA of host cells. More than 30 patients have been treated for SCID, and more than 90 percent of those children have been cured of their disorder-an improvement over the 50 percent chance of recovery offered by bone marrow transplants.

A risk involved with retroviruses is that they may stitch their gene anywhere into DNA, disrupting other genes and causing leukemia. Unfortunately, five of the 30 children treated for SCID have experienced this complication; four of those five, however, have beaten the cancer. Researchers are now designing delivery systems that will carry a much lower risk of causing this condition.

Although there are currently no gene therapy products on the market in the U.S., recent studies in both Parkinson's disease and Leber congenital amaurosis, a rare form of blindness, have returned very promising results. If these results are borne out, there could be literally hundreds of diseases treated with this approach.

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Gene therapy is the addition of new genes to a patient's cells to replace missing or malfunctioning genes. Researchers typically do this using a virus to carry the genetic cargo into cells, because that's what viruses evolved to do with their own genetic material.

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Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient's cells instead of using drugs or surgery. Researchers are testing several approaches to gene therapy, including:

Replacing a mutated gene that causes disease with a healthy copy of the gene.

Inactivating, or "knocking out," a mutated gene that is functioning improperly.

Introducing a new gene into the body to help fight a disease.

Although gene therapy is a promising treatment option for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections), the technique remains risky and is still under study to make sure that it will be safe and effective. Gene therapy is currently being tested only for diseases that have no other cures.

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What is gene therapy?: MedlinePlus Genetics

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Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient's cells instead of using drugs or surgery.

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Similar to cardiovascular diseases, gene therapy regarding lysosomal storage might be able to be cured with more of those "suicide genes", the problem right now is the genes don't last long, a human with the disease, when tested, will only hold the progression of the disease for 96 weeks for now.

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The item in question here is something called "suicide genes" that serve their use and get destroyed shortly after. With this they could engineer is DNA with the resistance or cure for a disease. Using the gene, they could target and destroy it.

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Gene therapy can cure only certain cardiovascular diseases that can be seen at birth (or at least have a risk to see). The disease that is being most prominently is glycogen storage disease type II. The disease almost stops energy storage and causes the heart to fail very early in life.

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The first treatment for cancer was in 1891, where a surgeon known as William Coley used bacteria to kill the cancer, and then a heating toxin to kill the bacteria. It did actually work. Which later was replaced by Radiation therapy and Chemotherapy which had slightly more consistent effects. The part that gene therapy comes in is experimental right now. It may use tumor-killing viruses which destroy the tumor and the inserted gene itself.

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In December 2017, the FDA approved Voretigene Neparvovec, or Luxturna, It is a one-time use gene therapy that improves vision for people such as me. However, it cannot cure things like colorblindness, but the researchers are planning on working on it in the future.

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Of course there is limits to how much it can do, not saying it does little, but a list can be much appreciated.
-Inherited retina disorders
-Cancer and Immunotherapies
-Cardiovascular diseases
-Infectious diseases
-Lysosomal Storage diseases
-Inherited Immunodeficiencis
-Musculoskeletal diseases
-Movement disorders
-Neurosensory diseases
-Red blood cell disorders
-Respiratory disorders
More on literally all of this ahead. (I'm only going to talk about half of them though.)

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The base of gene therapy is to correct errors with the gene pool. However, it's mostly experimental, but it has big plans.