Patient Perspectives on Gene Transfer Therapy for Sickle Cell Disease
نویسندگان
چکیده
منابع مشابه
Gene Therapy in a Patient with Sickle Cell Disease.
Sickle cell disease results from a homozygous missense mutation in the β-globin gene that causes polymerization of hemoglobin S. Gene therapy for patients with this disorder is complicated by the complex cellular abnormalities and challenges in achieving effective, persistent inhibition of polymerization of hemoglobin S. We describe our first patient treated with lentiviral vector-mediated addi...
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the risk of sickle cell complications that is a common hemoglobin disorder in Southwest Iran. This study aimed at determining the incidence of Sickle Cell Disease (SCD) and other Hemoglobinopathies in newborn being at risk based on ethnic origin. Materials and Methods: In this descriptive epidemiologic study, between September 2013 and September 2015, 8363 newborn blood samples were test...
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HLA-identical sibling hematopoietic cell transplantation (HCT) for sickle cell disease (SCD) has a strong track record of efficacy and there is growing appreciation that its benefits exceed its risks in selected individuals. In contrast, the clinical utility of replacement gene therapy for sickle cell disease remains unproven. Its challenge is to ensure viral transduction into hematopoietic ste...
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In patients with sickle cell disease, thrombotic microangiopathy is a rare complication. Also in sickle cell disease, intracardiac thrombus formation without structural heart diseases or atrial arrhythmias is a rare phenomenon. We herein describe a 22-year-old woman, who was a known case of sickle cell-βthalassemia, had a history of recent missed abortion, and was admitted with a vaso-occlusive...
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Background: Sickle cell anemia (SCA) is an inherited monogenic disorder. The clinical symptoms of SCA are protean, including vaso-occlusion, hemolysis, early stroke, leg ulcers, multi-organ failure, and increased risk of premature death. Hematopoietic stem cell transplantation is the only treatment identified to reduce SCA-related organ damage. Unfortunately, graft rejection is a significant im...
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ژورنال
عنوان ژورنال: Advances in Therapy
سال: 2017
ISSN: 0741-238X,1865-8652
DOI: 10.1007/s12325-017-0587-7