標題: Titlebook: Gene and Cell Therapies for Beta-Globinopathies; Punam Malik,John Tisdale Book 2017 Springer Science+Business Media LLC 2017 Beta-Globinop [打印本頁] 作者: Anagram 時間: 2025-3-21 19:19
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作者: Insatiable 時間: 2025-3-21 20:43
https://doi.org/10.1007/978-1-4939-7299-9Beta-Globinopathies; Hemoglobinopathies; cell therapy; gene therapy; thalassemias作者: Coronation 時間: 2025-3-22 02:00 作者: Anguish 時間: 2025-3-22 06:42
Gene and Cell Therapies for Beta-Globinopathies978-1-4939-7299-9Series ISSN 0065-2598 Series E-ISSN 2214-8019 作者: 反叛者 時間: 2025-3-22 11:01 作者: 配置 時間: 2025-3-22 15:10 作者: 配置 時間: 2025-3-22 19:07
https://doi.org/10.1007/978-3-7091-6653-6arose as common, balanced polymorphisms during human history because they afforded protection against severe forms of malaria. These complex, multisystem diseases are reviewed here with a focus on current standards of clinical management and recent research findings. The importance of a comprehensiv作者: enmesh 時間: 2025-3-22 22:29 作者: 水獺 時間: 2025-3-23 01:34
The Hypothalamo-Neurohypophyseal System,atched sibling donors in the 1980s, matched unrelated donors and cord blood sources in the 1990s, and haploidentical donors in the 2000s. Many studies have solidified hematopoietic progenitors from matched sibling marrow, cord blood, or mobilized peripheral blood as the best source—with the lowest g作者: BACLE 時間: 2025-3-23 07:11 作者: 使入迷 時間: 2025-3-23 13:06
Clinical Epidemiology and Biostatisticsions in the β-globin gene, such as β-thalassemia and sickle cell disease (SCD), since increasing the production of HbF can compensate for underproduction of β-globin chains (in β-thalassemia) and it can also disrupt sickle hemoglobin polymerization (in SCD). Thus for the past few decades, concerted 作者: brother 時間: 2025-3-23 15:59
Hepatitis B Virus: Asian Perspective, with α-globin, heme, and iron) of hemoglobin, the molecule essential for oxygen delivery to tissues, mutations in . can result in lethal diseases or diseases with multi-organ dysfunction. . mutations can be roughly divided into two categories: those that cause a dysfunctional protein (such as sickl作者: 彎腰 時間: 2025-3-23 22:05 作者: phase-2-enzyme 時間: 2025-3-23 22:14
Current Standards of Care and Long Term Outcomes for Thalassemia and Sickle Cell Disease,tem diseases are reviewed here with a focus on current standards of clinical management and recent research findings. The importance of a comprehensive, multidisciplinary and lifelong system of care is also emphasized.作者: 認識 時間: 2025-3-24 03:31 作者: 文字 時間: 2025-3-24 08:18 作者: Explosive 時間: 2025-3-24 13:37
Book 2017early 2% of the world’s population carries a globin gene mutation. The transfer of the corrective globin gene through the HSC compartment by allogeneic HSC transplantation (HSCT) has already proven curative in both SCD and thalassemia patients, and provides the proof of concept that genetic manipula作者: 愛花花兒憤怒 時間: 2025-3-24 16:24
Luke B. Hesson,Antonia L. Pritchard at a remarkable pace and great promise, many roadblocks remain before clinical translation can be realistically considered. Here we discuss the theoretical advantages of cell therapies utilizing hPSC derivatives, recent proof-of-principle studies and the main challenges towards realizing the potential of hPSC therapies in the clinic.作者: 包租車船 時間: 2025-3-24 21:48 作者: 好開玩笑 時間: 2025-3-25 01:55 作者: Musket 時間: 2025-3-25 05:58
,Genome Editing for the β-Hemoglobinopathies,e cell disease but also including varied diseases caused by high-affinity hemoglobins, low-affinity hemoglobins, and methemoglobinemia) and those that cause the insufficient production of HBB protein (β-thalassemia). Sickle cell disease and β-thalassemia are both the most prevalent and the most devastating of the β-hemoglobinopathies.作者: 上下倒置 時間: 2025-3-25 08:06
https://doi.org/10.1007/978-1-4613-8826-5 score to inform prognosis and guide management requires a larger panel of genetic modifiers yet to be discovered..Nonetheless, genetic studies have been successful in characterizing some of the key variants and pathways involved in HbF regulation, providing new therapeutic targets for HbF reactivation.作者: 機構(gòu) 時間: 2025-3-25 14:23
,Genetic Basis and Genetic Modifiers of β-Thalassemia and Sickle Cell Disease, score to inform prognosis and guide management requires a larger panel of genetic modifiers yet to be discovered..Nonetheless, genetic studies have been successful in characterizing some of the key variants and pathways involved in HbF regulation, providing new therapeutic targets for HbF reactivation.作者: Irritate 時間: 2025-3-25 19:12
,Clinical Features of β-Thalassemia and Sickle Cell Disease, 5–7% of the world’s population is a carrier of a significant hemoglobin variant. Without early diagnosis followed by initiation of preventative and therapeutic care, both SCD and β-thalassemia result in significant morbidity and early mortality. Despite great strides in the understanding of the mol作者: 用手捏 時間: 2025-3-25 22:02
,Genetic Basis and Genetic Modifiers of β-Thalassemia and Sickle Cell Disease,ne. Despite the apparent genetic simplicity, both disorders display a remarkable spectrum of phenotypic severity and share two major genetic modifiers—α-globin genotype and innate ability to produce fetal hemoglobin (HbF, α.γ.)..This article provides an overview of the genetic basis for SCD and β-th作者: 榨取 時間: 2025-3-26 02:03
Current Standards of Care and Long Term Outcomes for Thalassemia and Sickle Cell Disease,arose as common, balanced polymorphisms during human history because they afforded protection against severe forms of malaria. These complex, multisystem diseases are reviewed here with a focus on current standards of clinical management and recent research findings. The importance of a comprehensiv作者: anticipate 時間: 2025-3-26 06:47
,Allogeneic/Matched Related Transplantation for β-Thalassemia and Sickle Cell Anemia,alignant diseases have in common severe hemolytic anemia and high proliferative bone marrow, requiring frequent transfusions. The risk of rejection is high and graft-vs-host disease is not desirable. Important progress has been made in the management of these diseases, including leukocyte depletion 作者: Diuretic 時間: 2025-3-26 10:32
,Alternative Donor/Unrelated Donor Transplants for the β-Thalassemia and Sickle Cell Disease,atched sibling donors in the 1980s, matched unrelated donors and cord blood sources in the 1990s, and haploidentical donors in the 2000s. Many studies have solidified hematopoietic progenitors from matched sibling marrow, cord blood, or mobilized peripheral blood as the best source—with the lowest g作者: 敵意 時間: 2025-3-26 15:41
,Gene Addition Strategies for β-Thalassemia and Sickle Cell Anemia, is mutated, causing severe anemia and ineffective erythropoiesis. Patients can additionally present with a number of life-threatening co-morbidities, such as stroke or spontaneous fractures. Current treatment involves transfusion and iron chelation; allogeneic bone marrow transplant is the only cur作者: 潛移默化 時間: 2025-3-26 17:10 作者: browbeat 時間: 2025-3-26 21:26 作者: 控制 時間: 2025-3-27 02:59
,Gene and Cell Therapy for β-Thalassemia and Sickle Cell Disease with Induced Pluripotent Stem Cellsc engineering technologies, have opened new frontiers for cell and gene therapy. The prospect of using hPSCs, either autologous or histocompatible, as targets of genetic modification and their differentiated progeny as cell products for transplantation, presents a new paradigm of regenerative medici作者: 色情 時間: 2025-3-27 05:56
Book 2017 which are often available only to 15-20% of patients. An alternative to allogeneic HS.CT is genetic correction of autologous HSCs, to overcome donor availability & immune side effects. This Book reviews the progress made on additive gene therapy approaches & the current state of the field. Finally,作者: 侵略 時間: 2025-3-27 11:37
,Clinical Features of β-Thalassemia and Sickle Cell Disease,ive treatment for SCD but less so for β-thalassemia, and does not represent curative therapy. As technology and use of cellular and gene therapies expand, SCD and thalassemia should be among the highest disease priorities.作者: 你正派 時間: 2025-3-27 13:56 作者: neologism 時間: 2025-3-27 19:55
,Alternative Donor/Unrelated Donor Transplants for the β-Thalassemia and Sickle Cell Disease,some of the unrelated cord transplant studies. Haploidentical donors have emerged in the last decade to have less GvHD; however, improvements are needed to increase the engraftment rate. Thus the decision to use unrelated cord blood units or haploidentical donors may depend on the institutional expe作者: Tempor 時間: 2025-3-27 23:23 作者: 言外之意 時間: 2025-3-28 05:27 作者: 立即 時間: 2025-3-28 10:14 作者: 有花 時間: 2025-3-28 13:20 作者: Osmosis 時間: 2025-3-28 17:02
The Hypothalamo-Neurohypophyseal System,some of the unrelated cord transplant studies. Haploidentical donors have emerged in the last decade to have less GvHD; however, improvements are needed to increase the engraftment rate. Thus the decision to use unrelated cord blood units or haploidentical donors may depend on the institutional expe作者: 發(fā)起 時間: 2025-3-28 21:35
https://doi.org/10.1007/978-94-009-0105-6cal trials underway to test the curative potential of some of these lentiviral vectors. This review will highlight the work done thus far, and present the challenges still facing gene therapy, such as genome toxicity concerns and achieving sufficient transgene expression to cure those with the most 作者: 戰(zhàn)勝 時間: 2025-3-29 01:03
Clinical Epidemiology and Biostatisticsgs or regulatory proteins, and in only a fraction of patients, and there is wide variation in individual response to these drugs or transcription factors. These studies highlight the importance for understanding the molecular mechanisms underlying hemoglobin switching so that future studies can be d
