Gene therapy is still an experimental technique that uses genes to treat or prevent disease. In the future, this technique will allow doctors to treat, inter alia, disorders 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 only being tested for the treatment of diseases that have no other cures.
There are several methods available to target specific cells in the body, like modified adeno / retroviruses, and just last year there was a major breakthrough in gene editing, the discovery/developement of CRISPR-Cas9, which provides better specificity, simplicity, speed and pricing than most methods used before. Still at present, it is not possible to render an entire body transgenic.
Supporting research and development of gene therapy methods will allow HIP a quick proceeding for the next step of in-vivo trials. There are many different ways to approach gene therapy, but the safest and most effective one is still to be discovered