Release date: 2016-08-24 Eliza O'Neill suffers from Shaflip syndrome, a rare disease that is only one in 70,000 Recently, the foreign media website reported a story called "miracle." The hero of the story is Eliza O'Neill, a 6-year-old girl. Although seemingly naive, Eliza suffers from a deadly rare disease, Sanfilippo syndrome. As a disease with a probability of only one in 70,000, it causes children to lack a key enzyme that breaks down long-chain sugar molecules, which causes excessive accumulation of oligosaccharides in the body, causing damage to the body and hindering intelligence. development. Some people call it "children with Alzheimer's disease. " Most children do not live adolescents. To make matters worse, Eliza suffers from the most severe subtype A. The family’s will not leave Eliza is with her mother Cara, father Glenn and brother Beckham. In 2013, Eliza was diagnosed. In order to save her, the O'Neill family never gave up trying. They noticed that the research team had already performed gene therapy on a diseased mouse model, but they did not have enough funds to put it into clinical trials. Therefore, the O'Neill family raised more than $1.8 million through the “Save Eliza†campaign to encourage them to conduct clinical trials. However, no one can guarantee that Eliza will be included in this clinical trial. In order to gain a chance, O'Neill's family retired in 2014, only to avoid the immunity of Eliza to the virus used in gene therapy. Once immune to the treatment of the virus, Eliza will not be able to participate in the clinical trial. This "out of the box" has been in place for nearly two years. In 2 years, while everyone was looking forward to it, Eliza's illness became more and more obvious: at first, she was able to sing and speak complete sentences. Slowly, she can only speak 1 or 2 words, and eventually she can no longer express it... This is absolutely devastating. First-line vitality: gene therapy In May of this year, Eliza received gene therapy at the Children's Hospital of Columbus The virus used in gene therapy is similar to a cargo truck. It carries a key gene that is injected into the patient's body, allowing the cell to express key enzymes that are congenitally missing. On May 10, 2016, a team of doctors at the Children's Hospital of Columbus, Ohio, injected this harmless virus into the blood of Eliza. The 30-minute injection process is short and long, but full of desire for life. Fortunately, Eliza has been consistently stable during the treatment. Eliza's father, Glenn O'Neill, said: "Now we are full of hope for Eliza. I began to dare to think about her future." It should be emphasized that gene therapy is not perfect, not to mention that it is still in the clinical stage and requires a lot of work to verify its safety and effectiveness. Eliza was the first child to participate in the trial. So no one knows the ending. But the good news is that, at least so far, Eliza has been responding well. Mother Cara said: "Now, she will use her eyes to communicate with us and try to speak. We have seen the brilliance in her eyes again." Eliza parents said that now there is new hope The O'Neills family hopes to continue to raise more funds to help other children with Shaffei syndrome, giving them the opportunity to participate in clinical trials and improve. Finally, Dad Glenn said: "Eliza's future is still a lot uncertain. But if she does not receive treatment, her future can only be pain, epilepsy, nasal feeding, wheelchairs and death. So, uncertainty is a good thing because there is hope. !" (Note: The article was compiled from "Saving Eliza: Gene therapy offers new hope for girl with 'childhood Alzheimer's'".) Source: Bio-Exploration YT-88 YT-88 Shenzhen Sunshine Technology Co.,Ltd , https://www.shenzhenyatwin.com
