单词 | factor IX |
例句 | The gene codes for a protein involved in blood clotting called factor IX, which people with the disease are unable to produce. $3.5-Million Hemophilia Gene Therapy Is World’s Most Expensive Drug 2022-12-09T05:00:00Z At one point, Elliott was having injections of factor IX every other day in order to prevent a deadly bleed. Transformational therapy cures haemophilia B 2022-07-20T04:00:00Z A treatment for hemophilia B that restores a missing protein called factor IX, developed by the company BioMarin, is close to winning U.S. regulatory approval. Liver tumor in gene therapy recipient raises concerns about virus widely used in treatment 2020-12-22T05:00:00Z Now levels of factor IX in Jay Konduros’s blood are around 50 percent. They Thought Hemophilia Was a ‘Lifelong Thing.’ They May Be Wrong. 2018-08-13T04:00:00Z What’s more, eight of the 10 no longer need factor IX injections every few days. Gene therapy stops bleeding episodes in hemophilia trial 2017-12-06T05:00:00Z But scientists worry that the price would not be affordable in low- and middle-income countries, where most people with haemophilia live and where supplies of treatments and factor IX are often insufficient. $3.5-Million Hemophilia Gene Therapy Is World’s Most Expensive Drug 2022-12-09T05:00:00Z Elliot was given an engineered virus that was filled with the instructions for manufacturing the missing factor IX. Transformational therapy cures haemophilia B 2022-07-20T04:00:00Z UniQure’s hemophilia B treatment also appears to be working, with 52 of 54 patients no longer needing injections of factor IX after 6 months in its latest study. Liver tumor in gene therapy recipient raises concerns about virus widely used in treatment 2020-12-22T05:00:00Z Neither has required any factor IX since their gene therapy. They Thought Hemophilia Was a ‘Lifelong Thing.’ They May Be Wrong. 2018-08-13T04:00:00Z Gene therapy is showing promise for several rare diseases, including hemophilia B, in which people lack a livermade protein called factor IX that helps blood clot. How a liver-damaging drug may give a boost to gene therapy 2016-06-08T04:00:00Z Even the lowest response in the clinical trial, a 10% increase in factor IX levels, is sufficient to prevent spontaneous bleeding, researchers say. $3.5-Million Hemophilia Gene Therapy Is World’s Most Expensive Drug 2022-12-09T05:00:00Z He recalls being "astonished" to see the amount of factor IX in his blood go from only 1% of normal levels to normal. Transformational therapy cures haemophilia B 2022-07-20T04:00:00Z The team found that the monkey’s livers began producing much more factor IX: the protein's levels in the blood increased by 10%. Leukaemia success heralds wave of gene-editing therapies 2015-11-04T05:00:00Z Patients in Spark’s hemophilia B trial only reached on average 35 percent of normal blood levels of factor IX. But those levels have remained steady for the two years they have been followed. They Thought Hemophilia Was a ‘Lifelong Thing.’ They May Be Wrong. 2018-08-13T04:00:00Z So far, it hasn’t given hemophilia B patients enough factor IX to completely avoid bleeding episodes—they need about 12% of normal levels, but get half or less with the therapy. How a liver-damaging drug may give a boost to gene therapy 2016-06-08T04:00:00Z But patients might require top-up prophylaxis treatments after injuries, or if they’re having major surgery and their factor IX levels are less than 50%. $3.5-Million Hemophilia Gene Therapy Is World’s Most Expensive Drug 2022-12-09T05:00:00Z The findings, published in the New England Journal of Medicine, showed nine out of 10 patients given the therapy no longer needed their clotting factor IX injections. Transformational therapy cures haemophilia B 2022-07-20T04:00:00Z The procedure boosted levels of factor IX from zero to 1% of normal—enough to fuel optimism that a more efficient procedure might one day be possible. In the Fight against Haemophilia, Dogs are a Weapon 2015-01-19T05:00:00Z They treated ten patients with hemophilia B and managed to increase their blood levels of factor IX to between 2 percent to 6 percent of normal. They Thought Hemophilia Was a ‘Lifelong Thing.’ They May Be Wrong. 2018-08-13T04:00:00Z In 2010, they showed that oral delivery of factor IX expressed in chloroplasts in this way led to almost undetectable inhibitor levels in mice, and no sign of anaphylactic shock. Lettuce Pills (You Heard That Right) May Help Treat Haemophilia 2015-01-07T05:00:00Z As a result of an inherited genetic mutation, people with haemophilia B lack a protein called factor IX that is crucial for forming blood clots. The Search for Genetic Treatments for Haemophilia is Proving Difficult 2014-12-29T05:00:00Z In 2010, they showed that oral delivery of factor IX expressed in chloroplasts in this way led to almost undetectable inhibitor levels in mice, and no sign of anaphylactic shock1. Immunology: Oral solutions : Nature : Nature Publishing Group 2014-11-25T05:00:00Z DNA with a functional factor IX gene was bundled into the molecular wrapper of a virus — known as AAV8 — then shuttled into liver cells, where factor IX is normally made. Gene therapy: Genie in a vector : Nature : Nature Publishing Group 2014-11-25T05:00:00Z They found a man in Padua, Italy, who had a genetic mutation that made cells churn out as much as 12 times the usual amounts of factor IX. They Thought Hemophilia Was a ‘Lifelong Thing.’ They May Be Wrong. 2018-08-13T04:00:00Z Phase I safety studies of factor IX fused to albumin showed a fivefold increase in half-life, up to about four days. Clotting factors: Stretching time : Nature : Nature Publishing Group 2014-11-25T05:00:00Z The procedure boosted levels of factor IX from zero to 1% of normal — enough to fuel optimism that a more efficient procedure might one day be possible. Animal models: Dogged pursuit : Nature : Nature Publishing Group 2014-11-25T05:00:00Z Alprolix is a bioengineered version of the blood coagulation protein known as factor IX, which is needed for normal blood clotting. Exclusive: Biogen prices hemophilia drug on par with older therapies 2014-04-18T17:04:29Z Hemophilia A is caused by a lack of clotting factor VIII, while hemophilia B is caused by a lack of clotting factor IX. New hemophilia drugs aim to simplify treatment 2014-03-07T12:06:46Z “Our first patient was a 23-year-old nurse. His level of factor IX rose to around 30 percent and has remained there for two years,” she said. They Thought Hemophilia Was a ‘Lifelong Thing.’ They May Be Wrong. 2018-08-13T04:00:00Z In particular, work that is stretching the lifetime of factor IX by three to five times is “really transformative”, he says. Clotting factors: Stretching time : Nature : Nature Publishing Group 2014-11-25T05:00:00Z Haemophilia B is caused by mutations in the gene that codes for the factor IX protein; the treatment uses viruses to deliver a healthy version of the gene to patients' liver cells. Seven days: 9?15 December 2011 2011-12-14T18:20:20.353Z The two main forms of the disease are hemophilia A, caused by a lack of clotting factor VIII, and hemophilia B, caused by a lack of clotting factor IX. Gene therapy proves effective for hemophilia B 2011-12-11T03:11:57Z Patients in the study, whose bodies produced less than 1 percent of the normal amount of clotting factor IX, were getting injections of the standard treatment. Gene Therapy Allows Hemophiliacs to Skip Weekly Shots in Study 2011-12-10T17:35:08Z The nurse has not needed to inject factor IX and has had no bleeds, she added. They Thought Hemophilia Was a ‘Lifelong Thing.’ They May Be Wrong. 2018-08-13T04:00:00Z The only real difference is between factor IX, for which the techniques are offering extensions long enough to make a substantial difference in treatment, and factor VIII, for which the improvement has been more modest. Clotting factors: Stretching time : Nature : Nature Publishing Group 2014-11-25T05:00:00Z The two main forms of the disease are hemophilia A, caused by a lack of clotting factor VIII, and the less-common hemophilia B, caused by a lack of clotting factor IX. Biogen to build blood disorder drug franchise 2011-03-04T16:42:26Z Researchers have so far treated six men with severe hemophilia B who were producing clotting factor IX at less than 1 percent of normal levels. Gene therapy proves effective for hemophilia B 2011-12-11T03:11:57Z Shima has created an antibody with one arm that binds to factor IX and the other to factor X, pulling the two together so that the clotting cascade can continue. Clotting factors: Stretching time : Nature : Nature Publishing Group 2014-11-25T05:00:00Z And because half-lives can vary between patients, “at high doses, you could probably in some individual cases get a month's worth of factor IX,” Pipe says. Clotting factors: Stretching time : Nature : Nature Publishing Group 2014-11-25T05:00:00Z Only about 4% of people with haemophilia B develop inhibitors to factor IX. Clotting factors: Stretching time : Nature : Nature Publishing Group 2014-11-25T05:00:00Z The general goal of current treatment with recombinant factor IX is to achieve factor levels greater than 1 percent of normal. Gene therapy proves effective for hemophilia B 2011-12-11T03:11:57Z |
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