Gene therapy, the science of using genes to prevent or treat diseases, is poised to change healthcare in the country and around the world. Instead of merely treating the symptoms associated with complex diseases, gene therapy seeks to target the cause of the disease at the most basic level: genes and the mutation of those genes.
Gene therapy is not new; it has been studied for nearly half a century. However, exciting advances have occurred in the last few years, advances that are accelerating research and the availability of gene therapy.
What is gene therapy? At its most basic, gene therapy is treatment targeted at the mutated genes responsible for disease. There are three main approaches to gene therapy:
Scientists introduce genes into cells via a delivery vehicle called a vector, which is injected or passed through an IV into the specific tissue or area that’s being treated. Since the use of gene therapy within the human population is relatively new, it poses some unknown risks. Researchers are taking all steps to ensure the safety of gene therapy and thoroughly review all potential treatments before making them available to the public. Possible side effects include immune system reaction, infection and tumor formation.
Several companies are at the forefront of gene therapy research. While some are privately funded, many are funded through government grants. Companies pioneering gene therapy research include Spark Therapeutics, Alnylam Pharmaceuticals, Inc., Integrated DNA Technologies,bluebird bio, Myriad Genetics, Inc. and Crispr Therapeutics.
Gene therapy companies hail from all corners of the globe and focus on various aspects of gene therapy. For example, Alnylam Pharmaceuticals, Inc. focuses on RNA interference therapies while Integrated DNA Technologies focuses on manufacturing synthetic genes.
Gene therapy has the potential to treat a wide array of conditions, including immune deficiency, hemophilia, blindness, leukemia, cystic fibrosis, sickle cell anemia, muscular dystrophy, Parkinson’s, heart disease, cancer, diabetes, Alzheimer’s, HIV and numerous others.
In many cases, gene therapy is more promising than mainstream treatments for these diseases. Some gene therapy treatments may even offer a cure for diseases, such as cancer, where none currently exists. What’s more, gene therapy has already been used to cure some complex diseases like blindness. Doctors have been able to improve vision in nearly blind patients via gene therapy.
There are 1,500 known diseases caused by a single mutated gene. If researchers focus on these diseases alone, the potential of gene therapy to improve human existence and quality of life is astounding. However, gene therapy has also been used in diseases caused by multiple gene mutations, which means the possibilities are endless. In the future, most diseases may be treated, at least partially, with gene therapy.
Gene therapy is a cutting-edge, exciting treatment that’s providing hope for those with hard-to-treat diseases, such as cancer, neurological disorders and blood disorders. To date, the FDA has approved a handful of gene therapy treatments. However, the treatments are very expensive and difficult to get. It is hoped that gene therapy will become more accessible in the not-so-distant future as it carves out a place in mainstream medicine.
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