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Fred Hutch researcher gets $12.9M for 'bubble boy,' sickle-cell anemia work

Dr. Rainer Storb wants to make transplants safer and more widely available for patients who already suffer from immune diseases, such as "bubble-boy" disease. Now, Storb will have $12.9 million to work on that research, thanks to a grant from the National Heart, Lung, and Blood Institute. “Current approaches at cell and gene therapy for lethal noncancerous diseases of the blood and immune systems have inherent toxicities that may affect patients for the rest of their lives,” Storb said in a…
Source: bizjournals.com Health Care:Hospitals headlines - Category: Hospital Management Authors: Source Type: news

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CONCLUSION: A high prevalence of iron overload in this patient population in Latin American countries indicates that a better diagnosis and management of iron overload is required in these countries. PMID: 29663858 [PubMed - as supplied by publisher]
Source: Hematology - Category: Hematology Tags: Hematology Source Type: research
Sickle cell disease (SCD) is an inherited hemoglobinopathy characterized by hemolytic anemia, frequent painful episodes, poor quality of life, end organ damage and a shortened lifespan. While the seminal event is the polymerization of the abnormal hemoglobin, the downstream pathophysiology of vaso occlusion (VOC) results from heterotypic interactions between the altered, adhesive sickle cell RBC ′s, neutrophils, endothelium and platelets. Ischemia reperfusion injury, hemolysis and oxidant damage all contribute to heightened inflammation and activation of the hemostatic system.
Source: Seminars in Hematology - Category: Hematology Authors: Source Type: research
CONCLUSIONS: HbA decline was increased among patients with sickle cell anemia with prior immunologic response to RBC antigens and decreased among those with prior splenectomy, demonstrating that recipient immunologic characteristics influenced the clearance of transfused RBCs. PMID: 29664198 [PubMed - as supplied by publisher]
Source: Transfusion - Category: Hematology Authors: Tags: Transfusion Source Type: research
Abstract In the 100 years since sickle cell anemia (SCA) was first described in the medical literature, studies of its molecular and pathophysiological basis have been at the vanguard of scientific discovery. By contrast, the translation of such knowledge into treatments that improve the lives of those affected has been much too slow. Recent years, however, have seen major advances on several fronts. A more detailed understanding of the switch from fetal to adult hemoglobin and the identification of regulators such as BCL11A provide hope that these findings will be translated into genomic-based approaches to the t...
Source: Annual Review of Genomics and Human Genetics - Category: Genetics & Stem Cells Authors: Tags: Annu Rev Genomics Hum Genet Source Type: research
Abstract Patients with sickle cell anemia (SCA) can acquire many biochemical abnormalities, including altered magnesium levels. However, the roles of magnesium in the pathogenesis and management of SCA need to be determined. The aim of this work was to evaluate magnesium levels among pediatric patients with SCA in Basra, Iraq. The study employed a case-control design and examined 87 patients with SCA (3-15 years old) who had attended the Basra Center for Hereditary Blood Diseases while in a steady state and 90 apparently healthy control subjects. Complete blood count, red blood cell (RBC), and serum magnesium...
Source: Biological Trace Element Research - Category: Biology Authors: Tags: Biol Trace Elem Res Source Type: research
CONCLUSION: Targeted newborn screening, based on the results of sickling test in pregnant women, would misdiagnose more than one of six sickle cell anemia newborns who would not benefit from early care. Cost-effectiveness studies of routine newborn screening for sickle cell anemia should lead to a better screening strategy in contexts where hemoglobin S and other hemoglobin defect genes coexist. PMID: 29625861 [PubMed - as supplied by publisher]
Source: Revue d Epidemiologie et de Sante Publique - Category: Epidemiology Tags: Rev Epidemiol Sante Publique Source Type: research
This study investigated associations between SNPs in genes encoding metabolizing drug enzymes and laboratory parameters in sickle cell anemia patients under hydroxyurea (SCA-HU+). We evaluated hematologic and biochemical parameters by electronic methods and SNPs by PCR-RFLP and multiplex PCR in 35 SCA-HU+ patients and 67 SCA-HU- patients. The HbS, total cholesterol, lactate dehydrogenase, aspartate aminotransferase, total bilirubin and fractions levels, and leukocyte, eosinophil, monocyte, and erythroblast counts were reduced in SCA-HU+ patients (p C/-1053C>T were higher in SCA-HU+ patients (p A, CYP2E1 -1293G>C/-105...
Source: Disease Markers - Category: Laboratory Medicine Tags: Dis Markers Source Type: research
Abstract Sickle cell disease (SCD) is a monogenetic disorder marked by hemolytic anemia and vaso-occlusive complications. The hallmark of SCD is the intracellular polymerization of sickle hemoglobin (HbS) after deoxygenation, and the subsequent characteristic shape change (sickling) of red cells. Vaso-occlusion occurs after endothelial activation, expression of adhesion molecules and subsequent adhesion of leucocytes and sickle erythrocytes to the vascular wall. Here we review how oxidative stress from various sources influences this process. Emerging evidence points towards a dominant mechanism in which innate im...
Source: Clinical Hemorheology and Microcirculation - Category: Hematology Authors: Tags: Clin Hemorheol Microcirc Source Type: research
Abstract Sickle cell anemia is characterized by a mutation resulting in the formation of an abnormal beta-hemoglobin called hemoglobin S. Hemoglobin S polymerizes upon deoxygenation, causing impaired red blood cell deformability and increased blood viscosity at equivalent hematocrits. Thus, sickle cell disease is a hemorheologic disease that results in various pathologic processes involving multiple organ systems including the lungs, heart, kidneys and brain. Red blood cell mechanics and the perturbations on blood flow-endothelial interaction underlie much of the pathology found in sickle cell disease. Transfusion...
Source: Clinical Hemorheology and Microcirculation - Category: Hematology Authors: Tags: Clin Hemorheol Microcirc Source Type: research
Abstract This review focuses on the contribution of abnormal blood rheology in the pathophysiology of sickle cell anemia (SCA). SCA is characterized by a reduction of red blood cell (RBC) deformability but this reduction is very heterogeneous among patients. Recent works have shown that patients with the lowest RBC deformability (measured by ektacytometry) have enhanced hemolysis and would be more prone to develop several complications such as priapism, leg ulcers and glomerulopathy. In contrast, patients with the highest deformability, and not under hydroxyurea therapy, seem to develop more frequently vaso-occlus...
Source: Clinical Hemorheology and Microcirculation - Category: Hematology Authors: Tags: Clin Hemorheol Microcirc Source Type: research
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