Modern Medicine: Gene Therapy Revolutions

The technology of gene therapy has long been viewed as a pragmatic way in which to erase disease, by revising people’s DNA.

Gene therapy, as defined by the U.S. Food and Drug Administration, is a treatment in which a kind of replacement gene is added and integrated into a person’s body—or a disease-causing gene is inactivated. The process involves the addition of new instructions to cells, via billions of viruses with correct DNA strands.

The procedure is complex, first tested in 1990 with an abundance of negative side effects. While the past two decades have seen immense progress, gene treatments run at exorbitantly expensive prices.

Yet scientists and biotechnology entrepreneurs have continued to work and funnel money and resources into gene therapy, and 2016 has seen further growth and development. Italian scientists at Milan’s San Raffaele Telethon Institute for Gene Therapy reported that they had cured 18 children of a rare, extremely debilitating immune deficiency disease—ADA-SCID—by removing the children’s bone marrow and adding a gene to make the ADA enzyme that their bodies lacked.

Moreover, although the revolutionary cancer treatment that uses gene engineering to reprogram immune cells is not always considered a form of gene therapy, this type of immunotherapy has been proven to destroy certain types of cancer.

There is an abundance of promising results through human tests and studies, and 2017 will likely be the year in which the FDA evaluates and assesses several gene therapies. These include a treatment for hereditary blindness; approval would be an enormous breakthrough moment for the biotech industry—and one of the most inventive and pioneering ways to fully eradicate disease.