Gene therapy

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.

How gene therapy works
Gene therapy is designed to introduce genetic material into cells to compensate for abnormal genes or to make a beneficial protein. If a mutated gene causes a necessary protein to be faulty or missing, gene therapy may be able to introduce a normal copy of the gene to restore the function of the protein.

A gene that is inserted directly into a cell usually does not function. Instead, a carrier called a vector is genetically engineered to deliver the gene. Certain viruses are often used as vectors because they can deliver the new gene by infecting the cell. The viruses are modified so they can't cause disease when used in people.

CRISPR/Cas9
With CRISPR/Cas9, scientists can go into cells and edit the genes themselves more easily and cheaper than previous methods. These changes in the DNA will not only be transfered to the human that grows up, but their children as well.