![]() is currently studying the safety and tolerability of non-integrating gene therapy in people with CF. The study indicated that the CF gene therapy was safe and resulted in a small improvement in lung function. In a clinical trial in England, people with CF were given a dose of a non-integrating gene therapy once per month for a year. Food and Drug Administration to treat a rare type of blindness, and it has also been shown to work in studies for hemophilia, a blood clotting disorder. Non-integrating gene therapy has been approved by the U.S. ![]() A person with CF would probably need to be treated with the gene therapy repeatedly for it to be effective. The effect of the gene therapy might last only for several weeks or months. A disadvantage of non-integrating gene therapy is that it is not permanent. That means that the risk of side effects, including cancer, is low. Even though the gene therapy does not become part of the genome, the cell can still use the new copy of the CFTR gene to make normal CFTR proteins.Ī major advantage of the non-integrating gene therapy approach is that it does not disrupt the rest of the genome, just like adding a new page right under the cover of a book would not disturb the contents of the rest of the book. This is like placing a new page between the covers of an existing book without permanently attaching it. In non-integrating gene therapy, a piece of DNA with a correct copy of the CFTR gene is provided to an individual's cells, but the DNA remains separate from the genome and is not permanent. Watch this animation to see how this might work. Although the mutant copies of the CFTR gene would still be there, the presence of the correct copies would give cells the ability to make normal CFTR proteins. Gene therapy is a process in which a new, correct version of the CFTR gene would be placed into the cells in a person's body. Although progress was initially slower than anticipated, scientific breakthroughs in the past 10 years have accelerated advances in gene therapy, also known as gene transfer or gene replacement. Since the discovery of the CFTR gene in 1989, scientists have been trying to find ways to correct the mutations in the gene that cause CF. Each of these defects leads to a cascade of problems that affect the lungs and other organs. In people with cystic fibrosis, mutations in the CFTR gene can result in no protein, not enough protein, or the protein being made incorrectly. When there is a mutation - or alteration - in the genetic instructions, the production of the CFTR protein may be affected. The cystic fibrosis transmembrane conductance regulator (CFTR) gene contains the instructions for making the CFTR protein.
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