A small-scale gene therapy trial involving several Canadian patients provides new hope for hemophiliacs. According to research through recombinant dog proteins, hemophilia is a fatal hereditary rare disease. Due to lack of coagulation factors or protein, hemophilia patients would suffer from long-term uncontrollable bleeding, and even mild trauma would cause bleeding.
A new gene therapy developed by Philadelphia Children's Hospital has produced very encouraging results. Preliminary studies have shown that single therapy of experimental therapy may be able to help patients with type B hemophilia lacking clotting factor IX. The therapy involves the use of a designed gene to replace the wrong gene of hemophiliacs. The designed gene is placed into an inactivated virus and then injected into the liver, where it helps the patient produce clotting factors and prevent bleeding. "This brings the potential for a one - time treatment to change the patient's life," said Dr. Lindsey George, Ph.D., of the Philadelphia Children's Hospital.
Scientists have found that after only one dose of treatment, nine patients who participated in the trial did not show any bleeding for up to a year. Although more and more studies are needed to confirm the benefits of the therapy, the researchers are encouraged. "It's really exciting from my point of view as a clinical researcher," Dr. George said.
Among the nine patients treated, four were Canadian. One of them is the 52-year-old Ontario Cambridge Bakery owner, John Konduros. As a lifelong hemophilia patient, he has been living in any fear of any collision, cuts or scratches that may cause internal bleeding, disability or even death.
"It affects every part of my life from I'm a child until now," he told CTV News, "If you've ever seen my childhood, you'll find that I have never been in a group with other kids together. I'm always hiding aside," Konduros said. If another child happens to hurt his legs when playing, then he would be absent in 2 or 3 weeks. Konduros has not had any risk of bleeding since it received experimental treatment about eight months ago. So far, the immune system of the two trial patients has responded to the treatment, but the scientists said there was no serious side effect. Konduros did not have any problems.
Dr. Jerry Teitel, MD, director of the hemophilia treatment center at St. Michael's Hospital in Toronto, collaborated with Philadelphia researchers as "a revolutionary therapy". Dr. Teitel said, "In the development of drugs to help more hemophilia in the process, the scientists have a long way to go. We need to show that the results are reliable in a large number of patients. And we need to show that the results can last for a long time, but not sure whether they are for a lifetime." By the way, Flarebio offers superior recombinant proteins including recombinant Cdh11 at good prices.
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