| 单词 | thalassemia |
| 例句 | It helps meet high demand in Greece caused by the prevalence of thalassemia, a genetic disorder whose carriers need regular blood transfusions. Antonín Kohout 2013-02-26T18:12:07Z We do have a small pilot study of wheatgrass and the blood disorder thalassemia. What is chlorophyll water? 2022-12-05T05:00:00Z In beta thalassemia, the defective gene leads to deficient levels of hemoglobin in red blood cells. Sickle-Cell Treatment Created With Gene Editing Wins U.K. Approval 2023-11-16T05:00:00Z The agency approved the therapy, which the companies have called Casgevy, for patients ages 12 or older with sickle cell disease or beta thalassemia. United Kingdom approves first-ever CRISPR treatment, a cure for sickle cell disease and beta thalassemia 2023-11-15T05:00:00Z In people with thalassemia, the genetic mutation can cause severe anemia. The world’s first gene therapy for sickle cell and thalassemia has been approved 2023-11-16T05:00:00Z Both sickle cell disease and thalassemia are caused by mistakes in the genes that carry hemoglobin, the protein in red blood cells that carry oxygen. UK becomes 1st country to approve gene therapy treatment for sickle cell, thalassemia 2023-11-16T05:00:00Z Patients with beta thalassemia often need a blood transfusion every few weeks of their lives. Casgevy: UK approves gene-editing drug for blood disorders 2023-11-16T05:00:00Z In the United States, Bluebird already has a gene therapy approved for beta thalassemia. Sickle-Cell Treatment Created With Gene Editing Wins U.K. Approval 2023-11-16T05:00:00Z The therapy is also being tested in the related blood disease beta thalassemia. His sickle cell disease brought agony. Gene therapy is bringing hope. 2023-04-28T04:00:00Z Zynteglo is remarkable as a scientific achievement in treating beta thalassemia, a blood disorder. Hepatitis C is a slow-moving killer that can be stopped. What's getting in the way? 2023-01-01T05:00:00Z In people with thalassemia, the genetic mutation can cause severe anemia and patients typically require blood transfusions every few weeks, and injections and medicines for their entire life. UK becomes 1st country to approve gene therapy treatment for sickle cell, thalassemia 2023-11-16T05:00:00Z In trials, 28 out of 29 sickle cell patients were free of severe pain and 39 of 42 beta thalassemia patients no longer needed blood transfusions for at least a year. Casgevy: UK approves gene-editing drug for blood disorders 2023-11-16T05:00:00Z Called Casgevy, the treatment is intended to cure sickle-cell disease and a related condition, beta thalassemia. Sickle-Cell Treatment Created With Gene Editing Wins U.K. Approval 2023-11-16T05:00:00Z She and her team had previously treated fetuses with thalassemia, a genetic disease caused by lack of hemoglobin. The Disease Took Zara, Then Sara. Could Ayla Be Saved? 2022-11-09T05:00:00Z For the estimated 1,500 Americans suffering from a blood disorder known as beta thalassemia, the Food and Drug Administration’s approval Aug. 17 of the drug Zynteglo was almost unalloyed good news. Column: This life-saving drug will cost a record $2.8 million. Who will pay the price? 2022-09-09T04:00:00Z The agency approved the treatment for patients with sickle cell disease and thalassemia who are 12 years old and over. UK becomes 1st country to approve gene therapy treatment for sickle cell, thalassemia 2023-11-16T05:00:00Z The treatment for sickle cell disease and beta thalassemia is the first to be licensed using the gene-editing tool known as Crispr, for which its discoverers were awarded the Nobel prize in 2020. Casgevy: UK approves gene-editing drug for blood disorders 2023-11-16T05:00:00Z The agency will decide on approval for beta thalassemia next year. Sickle-Cell Treatment Created With Gene Editing Wins U.K. Approval 2023-11-16T05:00:00Z Bluebird is testing a different product that uses the same method for sickle cell anemia, which is more common in the United States than thalassemia. News at a glance: New gene therapy, Europe’s drought, and a black hole’s photon ring 2022-08-24T04:00:00Z Many will address diseases suffered by vastly larger populations than those with beta thalassemia. Column: This life-saving drug will cost a record $2.8 million. Who will pay the price? 2022-09-09T04:00:00Z In the study for thalassemia, 39 out of 42 patients who got the therapy did not need a red blood cell transfusion for at least a year afterwards. UK becomes 1st country to approve gene therapy treatment for sickle cell, thalassemia 2023-11-16T05:00:00Z More than 1,000 people in the UK are affected by thalassemia, mainly those of Mediterranean, southeast Asian and Middle Eastern origin. Casgevy: UK approves gene-editing drug for blood disorders 2023-11-16T05:00:00Z Diseases like sickle cell anemia and thalassemia decrease the blood’s ability to deliver oxygen to tissues and its oxygen- carrying capacity. Biology for AP Courses 2022-06-09T00:00:00Z A patient has thalassemia, a genetic disorder characterized by abnormal synthesis of globin proteins and excessive destruction of erythrocytes. Anatomy and Physiology 2013-06-19T00:00:00Z “For a newborn with beta thalassemia to grow up not tied to a healthcare system and iron overload and transfusions is a really great thing.” Column: This life-saving drug will cost a record $2.8 million. Who will pay the price? 2022-09-09T04:00:00Z Speaking at a hematology conference, the researchers reported that out of 44 treated patients with beta thalassemia, 42 no longer needed regular blood transfusions. CRISPR, 10 Years On: Learning to Rewrite the Code of Life 2022-06-27T04:00:00Z One early-phase clinical trial employed an ex vivo gene-editing method to treat people with sickle cell disease or with a related blood disorder called beta thalassemia. The Definition of Gene Therapy Has Changed 2021-10-19T04:00:00Z Patients with thalassemia produce a high number of red blood cells, but these cells have lower-than-normal levels of hemoglobin. Biology for AP Courses 2022-06-09T00:00:00Z The first results from a small clinical trial aimed at treating sickle cell disease and a closely related disorder, called beta thalassemia, were published this past June. Four Success Stories in Gene Therapy 2021-10-20T04:00:00Z The median age of death of those with beta thalassemia, which is typically diagnosed within a few weeks of birth, is 37. Column: This life-saving drug will cost a record $2.8 million. Who will pay the price? 2022-09-09T04:00:00Z The company shut down its trials for sickle cell patients and those with another blood disorder, beta thalassemia, while its researchers sought to understand if the gene therapy was at fault. Sickle cell treatment not linked to cancer, researchers say 2021-03-10T05:00:00Z Some blood disorders, such as sickle-cell anaemia and beta thalassemia, fit the bill. After the Nobel, what next for Crispr gene-editing therapies? 2021-02-21T05:00:00Z The results, published in Cell in 2019, also included specific variants related to cardiovascular diseases in Africans, such as one previously linked to an inherited blood disorder called alpha thalassemia. Africans begin to take the reins of research into their own genomes 2021-02-04T05:00:00Z Beta thalassemia, which affects millions more, occurs when a different mutation causes someone’s body to produce less hemoglobin, the iron-rich protein that allows red blood cells to carry oxygen. Four Success Stories in Gene Therapy 2021-10-20T04:00:00Z Those with severe beta thalassemia are forced to become “tethered to the health care system,” and even to specific geographical areas with medical centers that can provide the needed care, according to the report. Column: This life-saving drug will cost a record $2.8 million. Who will pay the price? 2022-09-09T04:00:00Z With the fetal gene now active, the fetal protein restores missing hemoglobin in thalassemia. CRISPR and another genetic strategy fix cell defects in two common blood disorders 2020-12-05T05:00:00Z She is now taking part in trials on Crispr treatments of both sickle-cell disease and beta thalassemia conducted in Boston by Crispr Therapeutics in collaboration with Vertex Pharmaceuticals. After the Nobel, what next for Crispr gene-editing therapies? 2021-02-21T05:00:00Z Other risk factors that might increase the risk of severe illness include asthma, cerebrovascular disease, cystic fibrosis, hypertension, immunocompromised state, neurologic conditions, liver disease, pregnancy, pulmonary fibrosis, thalassemia and Type 1 diabetes. Being overweight now potential coronavirus risk factor, CDC says 2020-10-09T04:00:00Z Fifteen people with beta thalassemia were treated with CTX001; after three months or more, all 15 showed rapidly improved hemoglobin levels and no longer required blood transfusions. Four Success Stories in Gene Therapy 2021-10-20T04:00:00Z Initial attempts should be for serious diseases caused by a single gene, such as muscular dystrophy, cystic fibrosis, the blood disorder beta thalassemia and Tay-Sachs, a neurological disease, the report says. Still too soon to try altering human embryo DNA, panel says 2020-09-03T04:00:00Z He suffered from a rare kidney disorder, thalassemia, that required bi-monthly transfusions, an experience that coloured his work. 'Heroin chic’ and the tangled legacy of photographer Davide Sorrenti 2019-05-23T04:00:00Z Corrections & Amplifications There are an estimated 1,000 people in the U.S. with beta thalassemia. Biotech Proposes Paying for Pricey Drugs by Installment 2019-01-08T05:00:00Z In the past few years, gene-replacement techniques have advanced to the point where they can treat congenital blood disorders, such as hemophilia and thalassemia. The History of Blood 2019-01-07T05:00:00Z More than a year later those improvements persisted in five subjects with beta thalassemia and two with sickle cell. Four Success Stories in Gene Therapy 2021-10-20T04:00:00Z “It’s no more only about shortages in drugs for cancer or special diseases such as haemophilia or thalassemia, but also normal drugs that were abundant in Iran previously,” she said. 'A matter of life and death': Iranians despair as US sanctions bite 2018-08-07T04:00:00Z The condition, alpha thalassemia major, leaves red blood cells unable to carry oxygen around the body, causing severe anemia, heart failure and brain damage. Five Blood Transfusions, One Bone Marrow Transplant — All Before Birth 2018-05-25T04:00:00Z In 2017, researchers in China used base editing to mend mutations that cause a serious blood disorder called beta thalassemia in human embryos. Gene editing – and what it really means to rewrite the code of life 2018-01-15T05:00:00Z A company called Crispr Therapeutics, based in Cambridge, Massachusetts, will use Crispr to fix a genetic defect in patients with an inherited blood disorder called beta thalassemia. Forget Juicero, here are the tech gadgets we can actually get excited about in 2018 2017-12-28T05:00:00Z The first published work involving human embryos, reported in 2015, was done in China and targeted a gene that leads to the blood disorder beta thalassemia. First human embryo editing experiment in U.S. ‘corrects’ gene for heart condition 2017-08-02T04:00:00Z The first published human embryo–editing work, in 2015, used nonviable embryos and targeted a gene mutated in the heritable blood disorder beta thalassemia. First U.S. team to gene-edit human embryos revealed 2017-07-27T04:00:00Z Everything pointed toward alpha thalassemia major — the worst form of the disease. Five Blood Transfusions, One Bone Marrow Transplant — All Before Birth 2018-05-25T04:00:00Z In addition to being the first case of pre-pubertal freezing, her delivery is the first from a patient who underwent treatment for beta thalassemia, Picton said. Woman believed to be the first to have a baby using ovary frozen before puberty 2016-12-15T05:00:00Z Approval of the gene therapy paves the way for the development of treatments for more widespread illnesses such as thalassemia and sickle cell disease. Gene therapy drug approval granted to GSK - BBC News 2016-05-27T04:00:00Z Chinese scientists attempted to alter genes in human embryos that cause a blood disorder, beta thalassemia, in an experiment deemed ethical by a Chinese national committee because the embryos were not viable. A Pause to Weigh Risks of Gene Editing 2015-12-18T05:00:00Z Sangamo also plans to apply for permission to do several other in vivo gene-editing trials, including of therapies for the blood diseases haemoglobinopathy and beta thalassemia. Leukaemia success heralds wave of gene-editing therapies 2015-11-04T05:00:00Z Alpha thalassemia major involves two genes, and two flawed copies of each. Five Blood Transfusions, One Bone Marrow Transplant — All Before Birth 2018-05-25T04:00:00Z The conditions, apart from cystic fibrosis, include sickle cell anemia, Tay-Sachs disease and beta thalassemia. 23andME Launches New Consumer Test Service to Check for Genetic Disorders 2015-10-21T04:00:00Z Mr. Girondi accuses the cancer center of dawdling on developing a gene therapy that could potentially cure his son of an inherited blood disease called beta thalassemia, or Cooley’s anemia. Lawsuit Accuses Sloan Kettering of Delaying Gene Therapy for Rare Blood Disease 2015-10-15T04:00:00Z The health ministry scrambled for currency, but within months the shortage of medicine was critical for millions of Iranians suffering from multiple sclerosis, hepatitis, haemophilia, cancer, diabetes and thalassemia. Iran under sanctions: no money for medicine but luxury cars aplenty 2015-08-26T04:00:00Z The investigators tried to repair a mutation in a gene that causes a potentially fatal blood disorder known as beta thalassemia. Altering Embryo Genes, Safely, Should Not Be Off-Limits 2015-06-18T04:00:00Z The investigators used the Crispr method to try to edit a gene that, when mutated, causes beta thalassemia, a serious blood disorder. Chinese Scientists Edit Genes of Human Embryos, Raising Concerns 2015-04-23T04:00:00Z He suffers from thalassemia, a genetic blood disorder so serious it requires monthly transfusions for life. India's illegal market for blood 2015-01-27T05:00:00Z Bluebird, by contrast, has reported on the treatments for seven patients with beta thalassemia and one with the more common sickle cell anemia, which can also potentially be treated with the same therapy. Lawsuit Accuses Sloan Kettering of Delaying Gene Therapy for Rare Blood Disease 2015-10-15T04:00:00Z An estimated 50,000 Pakistanis suffer from thalassemia, which requires blood transfusions at least once a month. Pakistani Children Infected With H.I.V. From Transfusions, Report Says 2014-12-05T05:00:00Z Such conditions include cancers like leukemia, certain lymphomas, multiple myeloma and aplastic anemia; inherited disorders like sickle cell anemia and thalassemia; and severe immune deficiency disorders in newborns. Well: Lifesaving Procedure With an Image Problem 2014-04-21T04:01:41Z It also said such groups were more at risk of developing thalassemia, a group of inherited blood disorders that hindered the blood's ability to carry oxygen to the lungs. Plea for rare group blood donors 2014-03-14T15:40:56Z The Swiss blood shipped to Greece helps meet demand from the country's thalassemia sufferers. Swiss Red Cross cuts blood supply to broke Greece 2013-02-26T18:46:07Z He has told people, however, that he is frustrated that the beta thalassemia project has progressed so slowly. Lawsuit Accuses Sloan Kettering of Delaying Gene Therapy for Rare Blood Disease 2015-10-15T04:00:00Z Not long after his return, Karthik witnessed near his home in Bangalore a family’s struggle to find regular blood transfusions for their four-year-old daughter to treat her thalassemia, a genetic blood disease. A New Generation of Techonomists Are Driven To Change the World 2011-11-10T15:46:19Z Studying Tropical Genetic Blood Diseases Sir David Weatherall, 77, an Oxford researcher-physician, was among the first to use the tools of molecular biology to understand thalassemia. A Conversation With David J. Weatherall: Studying Tropical Genetic Blood Diseases 2010-10-12T15:20:00Z He was honored for 50 years of “international statesmanship in biomedical science,” including his research on an inherited anemia called thalassemia. Vision and Obesity Researchers Are Winners of Lasker Awards 2010-09-21T01:49:00Z Because of a genetic mutation, people with beta thalassemia do not properly produce hemoglobin, the protein in red blood cells that transports oxygen to the body’s tissues. Lawsuit Accuses Sloan Kettering of Delaying Gene Therapy for Rare Blood Disease 2015-10-15T04:00:00Z In 1992, he learned that his young son had beta thalassemia. Lawsuit Accuses Sloan Kettering of Delaying Gene Therapy for Rare Blood Disease 2015-10-15T04:00:00Z What was so important about our thalassemia research was that the techniques we pioneered became a kind of template for understanding genetic diseases. A Conversation With David J. Weatherall: Studying Tropical Genetic Blood Diseases 2010-10-12T15:20:00Z I used this time to search for more thalassemia. A Conversation With David J. Weatherall: Studying Tropical Genetic Blood Diseases 2010-10-12T15:20:00Z Sir David Weatherall, 77, an Oxford researcher-physician, was among the first to use the tools of molecular biology to understand thalassemia. A Conversation With David J. Weatherall: Studying Tropical Genetic Blood Diseases 2010-10-12T15:20:00Z |
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