Gene therapy targeting fetal fatal diseases is expected to minimize certain genetic disease damage August 02, 2018 Source: Chinese Journal of Science and Technology The US government last year considered whether to approve a gene therapy to prevent retinal cell degeneration. Image credit: P. Motta/Dept. of Anatomy/University “La Sapienzaâ€, Rome/SPL A study in embryonic mice has shown that gene therapy implemented in the uterus can be used to treat a deadly genetic disease. Related results may add more evidence to the concept of the increasingly popular prenatal gene therapy. This therapy is seen as a way to minimize the damage caused by certain genetic diseases. Last year, the US Food and Drug Administration approved the first gene therapy for adults and children. Looking at the world, more therapies are emerging. Simon Waddington, the lead author of the latest study, said that when discussing the use of gene therapy to treat the fetus, he encountered shocked and angry eyes. “In fact, I have to give up telling people that fetal gene therapy is a good idea.†Waddington, who studies gene therapy at University College London, said, “Now, people often ask me, 'Do you know what a good idea is? Fetal gene therapy .'" The mouse study, published in the journal Nature Medicine, used prenatal gene therapy to treat acute neuropathic Gaucher disease. The disease is caused by a mutation in a gene called GBA. These mutations disrupt the breakdown of specific fat molecules, or lipids. As a result, lipids accumulate in the brain cells and other parts of the body, causing organ dysfunction. The study explored whether the disease could be treated with a virus that supplies a normal copy of GBA to a developing fetus. This may minimize irreparable brain damage caused by lipid accumulation. Some forms of Gaucher disease can be treated by decomposing lipids by supplying a normal copy of the GBA enzyme, but this enzyme cannot pass through the blood into the brain. Children with acute neuropathic Gaucher disease rarely survive two years of age. Waddington said the disease was so destructive that his colleagues expressed doubts about their team's ability to treat the disease. One obstacle is to let the virus carry healthy genes into the brain. The viruses used in the previous tests had to be injected directly into the brain, and then they spread only in close proximity to the injection site. But in 2009, researchers confirmed that a specific virus that can be injected into the blood can reach the central nervous system. From there, it spreads throughout the brain. Mice studied by Waddington were loaded with a virus that had a normal copy of GBA. He started with these mice looking for ways to make them specifically expressed in the central nervous system. The team tested the virus in embryonic mice. The mice carry a GBA mutation that causes similar symptoms to neuropathic Gaucher disease. Usually, such mice can only live for up to 15 days after birth, but this time they live for at least 18 weeks and can move around normally. Tippi MacKenzie, a specialist in fetal medicine at the University of California, San Francisco, said the work was impressive. MacKenzie has been conducting clinical trials of prenatal stem cell transplantation. “Fetal gene therapy or enzyme replacement therapy may be the next frontier,†she said. “It’s great to see this meticulous research. It takes us one step closer.†Gene therapy has several potential advantages for treating the fetus. The most important advantage is that the damage caused by hereditary diseases, such as neuropathic Gaucher disease and spinal muscular atrophy, can cause irreversible symptoms before the baby is born. At the same time, it is easier to perform some treatments on the developing fetal brain than adults or children, because the blood-brain barrier—the membrane that blocks some molecules from entering the brain—has not yet fully formed. “Even if it is one day after birth, it becomes more difficult to enter the brain,†said Duke, an obstetrician and gynecologist at the National University of Singapore. Since the fetal immune system is still developing, it is less likely to recognize newly expressed proteins as outsiders. Adult immune systems sometimes produce antibodies against new proteins that prevent them from performing functions. Chan and others have previously tested fetal gene therapy for hemophilia in mice and macaques. Chan hopes that people will be interested in the use of related therapies to treat several metabolic diseases similar to Gaucher disease. However, the risks also exist. Researchers who develop prenatal gene therapy should consider not only the fetus, but also the mother, who will inevitably receive treatment. Waddington said clinicians had to determine in advance that the mutations they found would cause disease. This may mean combining genetic testing with other tests performed in the womb to confirm the disease. (Zonghua compilation) Related paper information: DOI: 10.1038/d41586-018-05726-5 Chinese Journal of Science and Technology (2018-08-02 3rd Edition International) Pain Relief Patch For Neck Shoulder,Lower Back And Leg
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