Titlebook: Gene and Cell Therapies for Beta-Globinopathies; Punam Malik,John Tisdale Book 2017 Springer Science+Business Media LLC 2017 Beta-Globinop

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,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.

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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.

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,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.

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,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

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,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

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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

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,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

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,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

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,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

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