The Wide Angle: Top 10 Promising Gene TherapiesBy Vicki Miskovsky
The Cure Inside Us Treating the DNA through gene therapy could prove more effective than treating the disease with medicines that merely address the symptoms.
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Everything about the way the human body grows and functions is a result of genetic code. When the code is bad, when genes are defective, disorders and diseases can rear their ugly heads. Few of these diseases can be cured. But medical researchers around the world are working on genetic therapies that may mend genetic mistakes, replace flawed genes or supply missing ones. Gene therapy may also work by introducing special genes to kill cancer cells or to block the supply of blood to tumors. Some diseases, like cystic fibrosis or hemophilia, are the result of a defect on only one gene (single-gene genetic disorders) and this makes them good candidates for the use of gene therapy. Other diseases, like cancer, are more complicated to treat because several genes as well as life-style and environmental factors may cause the disease. There are abundant challenges in developing a human gene therapy. Even with single-gene disorders, it's difficult to deliver the corrective genes to the appropriate place in the body. Other challenges include the need to control the amount of time the therapeutic genes remain active; sometimes the corrective genes are needed for a lifetime, and other times they are needed only until they achieve their intended benefit, such as shrinking a tumor. As a result, gene therapies have not yet eliminated disease. But Donald Kohn, head of the Division of Research Immunology-Bone Transplantation, Children's Hospital, Los Angeles says, "One by one, we'll have the tools that are advantageous for various diseases." Gene therapy in the United States is available only through participation in a clinical trial. In 1992 there were 11 gene therapy trials. Today, if you visit the the gene therapy clinical trials website and search for "gene therapy" you'll find hundreds of gene therapy trials. Dr. Barrie J. Carter, President Elect of the American Society of Gene and Cell Therapy, believes that clinical trials in the last twenty years have shown the overall safety of gene therapy and predicts, "The next 20 years will show some spectacular successes." Here are 10 research gene therapies with potential, chosen for impact on otherwise untreatable diseases or for the treatment of diseases that affect a large number of people. |
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Want more? Click here for the rest of the Wide Angle: Genetic Science. Credit: Wikimedia Commons
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