With severe hemophilia B, all 10 men, after receiving gene therapy, Factor IX levels are elevated, and remained stable in more than four years. Overall, spontaneous bleeding episodes decreased by 90%. In the first 12 months after the investigation based therapy, the use of Factor IX replacement therapy reduced by approximately 92%.
Receiving the highest dose of 6 subjects, levels of coagulation proteins from normal level (less than 1%), increased by 5% or more. This increase makes them into a serious illness from mild to enable participants to participate in sports, such as football, without having Factor IX replacement therapy does not increase the risk of bleeding.
Receiving the highest dose of 6 patients, 4 patients with increased liver enzymes, may be due to an immune response to the carrier. These people have no symptoms or stay healthy, after Human Interleukin 6 ELISA Kit http://www.cusabio.com/html/product/CSB-E04638h.html, their liver enzymes returned to normal range.
Hemophilia B is a mutant Factor IX gene causes that can lead to low levels of coagulation proteins important risk. The disease affects about 30,000 individuals among a majority of men. The disease is characterized by Factor IX levels less than 1 percent of normal for these patients, scrapes and collisions will lead to medical emergencies. Spontaneous bleeding episodes of pain that can lead to severe joint damage early, increasing the risk of potentially fatal brain hemorrhage.
In this paper, the senior author Dr. Andrew Davidoff St. Jude Children's Research Hospital, Department of Surgery, said: "This study provides the first evidence that this gene therapy has long-term safety and efficacy so far, these results by dramatically reducing the risk of bleeding. of life study participants had a significant impact. "
In this paper, first author and corresponding author Amit Nathwani added: "We have reported data, marking a model of hemophilia B treatment changes, and for the treatment of bleeding disorders such major basis of these results, it is. development of such gene transfer methods, for the treatment of other diseases (from other congenital clotting defects such as hemophilia a, to metabolic birth defects, such as phenylketonuria), provides a solid platform. "
Before gene therapy, 70% of patients at least once a week to accept the Factor IX replacement therapy to prevent bleeding episodes. Others according to need replacement therapy to stop the bleeding.
Since participating in the trial, seven patients who had finished the regular four injections of Factor IX. Despite the increase of physical activity, but no one spontaneous bleeding. Nathwani said: "Some of the patients within four years of the time do not need clotting factor, which has changed their lives." Researchers estimate that these study participants accepted Factor IX replacement therapy overall spending more than $ 2.5 million .
Factor IX is usually produced by hepatocytes. AAV8 was selected as the carrier, because the virus can infect liver cells, but does not cause disease in humans or integrate into human DNA. This study is limited in patients with no previous AAV8 infection, to eliminate the need for immunosuppressive drug, such that the carrier of the immune system from possible attacks.
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