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PubMed Journals Articles About "Affordable Sickle Cell Disease Gene Therapies" RSS

03:24 EST 24th February 2020 | BioPortfolio

Affordable Sickle Cell Disease Gene Therapies PubMed articles on BioPortfolio. Our PubMed references draw on over 21 million records from the medical literature. Here you can see the latest Affordable Sickle Cell Disease Gene Therapies articles that have been published worldwide.

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Showing "Affordable Sickle Cell Disease Gene Therapies" PubMed Articles 1–25 of 51,000+

Multicenter Evaluation of HemoTypeSC as a Point-of-Care Sickle Cell Disease Rapid Diagnostic Test for Newborns and Adults Across India.

Sickle cell anemia is the commonest genetic disorder in India, and the frequency of the sickle cell gene is very high in the remote tribal areas where facilities are generally limited. Therefore, a rapid and affordable point-of-care test for sickle cell disease is needed.


Hematopoietic stem cell transplantation and cellular therapy in sickle cell disease: where are we now?

Sickle cell disease (SCD) is a common monogenic disorder that is characterized by an A to T substitution in the β-globin gene that leads to the production of hemoglobin S (HbS). Polymerization of HbS leads to significant morbidity including vaso-occlusion, pain, hemolytic anemia, and end organ damage. Allogeneic hematopoietic cell transplantation (allo-HCT) is the only curative treatment; however, suitable donors are not always readily available. This study reviews the current status of allo-HCT and autolo...

Preference-based measure of health-related quality of life and its determinants in sickle cell disease in Nigeria.

Health-related quality of life (HRQL) and economic burden are important issues for people with sickle cell disease (SCD) owing to better survival due to medical advances. Preference-based or utility information is necessary to make informed economic decisions on treatment and alternative therapies. This study aimed to assess preference-based measures of HRQL in sickle cell patients.


Association of the Δ Mutant Genotype with Sickle Cell Disease in Egyptian Patients.

Sickle cell disease is considered the most common single base mutation in the world, with >250,000 new patients being discovered each year. It consists of a wide spectrum of clinical presentations and complications. The is the mutant genotype of C-C chemokine receptor 5 (CCR5). It is widely distributed due to several micro organisms that target macrophages in different populations. Theoretically, confers an advantage to sickle cell disease patients. The chronic inflammatory response is the main pathogenes...

Web-Based Technology to Improve Disease Knowledge Among Adolescents With Sickle Cell Disease: Pilot Study.

Advancements in treatment have contributed to increased survivorship among children with sickle cell disease (SCD). Increased transition readiness, encompassing disease knowledge and self-management skills before transfer to adult care, is necessary to ensure optimal health outcomes. The Sickle Cell Transition E-Learning Program (STEP) is a public, Web-based, 6-module tool designed to increase transition readiness for youth with SCD.

Use of an automated pyrosequencing technique for confirmation of sickle cell disease.

The diagnosis of sickle cell disease (SCD) is made by hemoglobin assays such as high-performance liquid chromatography (HPLC), isoelectric focusing and cellulose acetate or citrate agar electrophoresis. These assays are easy to perform and used in large-scale newborn screening in many countries. These tests however may not easily differentiate Sβ0 thalassemia from SS or identify other hemoglobin variants, and in this case, hemoglobin (HBB) gene sequencing may be necessary.

The increased neopterin content in turkish pediatric patients with sickle cell anemia.

In the present study, the possible activation of cellular immunity in SCD patients was investigated. As immune activation parameters, neopterin concentrations and kynurenine/tryptophan ratio for tryptophan degradation in 35 pediatric patients with sickle cell disease (31 HbSS and 4 HbSß) were determined. Our results have shown that neopterin levels (both urinary and serum) are increased in pediatric patients with sickle cell disease. The increase in neopterin concentration was accompanied by significantly ...

Prevalence of enuresis and its impact in quality of life of patients with sickle cell disease.

Evidence indicates an increase in the prevalence of enuresis in individuals with sickle cell disease. The present study aims to evaluate the prevalence and impact of enuresis on quality of life in individuals with sickle cell disease.

Evaluation of Ocular Complications by Using Optical Coherence Tomography in Children With Sickle Cell Disease Eye Findings in Children With Sickle Cell Disease.

The aim of this study was to compare optical coherence tomography (OCT) findings in pediatric patients with sickle cell disease (SCD) and healthy individuals and to investigate associations between these data and the patients' systemic findings.

American Society of Hematology 2019 guidelines for sickle cell disease: cardiopulmonary and kidney disease.

Prevention and management of end-organ disease represent major challenges facing providers of children and adults with sickle cell disease (SCD). Uncertainty and variability in the screening, diagnosis, and management of cardiopulmonary and renal complications in SCD lead to varying outcomes for affected individuals.

Exploring the Needs of Adolescents With Sickle Cell Disease to Inform a Digital Self-Management and Transitional Care Program: Qualitative Study.

Accessible self-management interventions are critical for adolescents with sickle cell disease to better cope with their disease, improve health outcomes and health-related quality of life, and promote successful transition to adult health care services. However, very few comprehensive self-management and transitional care programs have been developed and tested in this population. Internet and mobile phone technologies can improve accessibility and acceptability of interventions to promote disease self-man...

Acute Care Utilization at End of Life in Sickle Cell Disease: Highlighting the Need for a Palliative Approach.

People with sickle cell disease (SCD) have a life expectancy of

Splenectomy is not associated with a higher tricuspid regurgitant jet velocity in people with sickle cell anemia.

Vascular complications such as pulmonary hypertension (PH) occur at an increased rate following splenectomy in patients with various hemolytic blood disorders including thalassemia. The goal of this retrospective cross-sectional analysis was to assess the independent association of splenectomy with an elevated tricuspid regurgitation velocity (TRV) in people with homozygous sickle cell disease (HbSS). TRV is a noninvasive screening test for PH and a surrogate marker of prognosis in sickle cell disease (SCD)...

Combined genetic disruption of K-Cl cotransporters and Gardos channel KCNN4 rescues erythrocyte dehydration in the SAD mouse model of sickle cell disease.

Excessive red cell dehydration contributes to the pathophysiology of sickle cell disease (SCD). The densest fraction of sickle red cells (with the highest corpuscular hemoglobin concentration) undergoes the most rapid polymerization of deoxy-hemoglobin S, leading to accelerated cell sickling and increased susceptibility to endothelial activation, red cell adhesion, and vaso-occlusion. Increasing red cell volume in order to decrease red cell density can thus serve as an adjunct therapeutic goal in SCD. Regul...

Biallelic correction of sickle cell disease-derived iPSCs confirmed at the protein level through serum-free iPS-sac/erythroid differentiation.

New technologies of induced pluripotent stem cells (iPSCs) and genome editing have emerged, allowing for the development of autologous transfusion therapies. We previously demonstrated definitive β-globin production from human embryonic stem cell (hESC)-derived erythroid cell generation via hemangioblast-like ES-sacs. In this study, we demonstrated normal β-globin protein production from biallelic corrected sickle cell disease (SCD) iPSCs. We optimized our ES/iPS-sac method for feeder cell-free hESC maint...

American Society of Hematology 2020 guidelines for sickle cell disease: transfusion support.

Red cell transfusions remain a mainstay of therapy for patients with sickle cell disease (SCD), but pose significant clinical challenges. Guidance for specific indications and administration of transfusion, as well as screening, prevention, and management of alloimmunization, delayed hemolytic transfusion reactions (DHTRs), and iron overload may improve outcomes.

Correlates of Cognitive Function in Sickle Cell Disease: A Meta-Analysis.

To provide a comprehensive quantitative review of biological, environmental, and behavioral correlates across domains of cognitive function in sickle cell disease (SCD).

Gene therapy in sickle cell disease: Possible utility and impact.

Sickle cell disease: A primary care update.

Sickle cell disease (SCD) is the most common hemoglobinopathy in the United States and causes significant disease-related morbidity including multiorgan damage, chronic anemia, and debilitating pain crises. Primary care physicians play a key role in the medical home model of care for adults with SCD. This review focuses on current recommendations for health maintenance and provides a brief summary of disease complications and current updates.

Reflections of Healthcare Experiences of African Americans With Sickle Cell Disease or Cancer: A Qualitative Study.

The experiences of African American adult patients before, during, and after acute care utilization are not well characterized for individuals with sickle cell disease (SCD) or cancer.

Current and emerging treatments for sickle cell disease.

Sickle cell disease (SCD) is a group of inherited blood disorders affecting the hemoglobin, shortening the lifespan of erythrocytes, and causing them to take on a distinctive sickled shape that can lead to vaso-occlusion. Current treatment aims to reduce morbidity and mortality through hydroxyurea, erythrocyte transfusion, and hematopoietic stem cell transplantation. This article reviews the disease process, typical presentations, complications, and acute and chronic treatment options.

A QST-based Pain Phenotype in Adults with Sickle Cell Disease: Sensitivity and Specificity of Quality Descriptors.

We sought to refine a screening measure for discriminating a sensitized or normal sensation pain phenotype among African American adults with sickle cell disease (SCD).

Retinal Thickness and Microvascular Changes in Children with Sickle Cell Disease Evaluated by Optical Coherence Tomography and Optical Coherence Tomography Angiography.

To compare the severity of macular vascular changes in children with sickle cell disease (SCD) versus age- and race-matched controls DESIGN: Cross-sectional study METHODS: Children (

Liver Stiffness Measurement by Vibration Controlled Transient Elastography Does Not Correlate to Hepatic Iron Overload in Children With Sickle Cell Disease.

Children with sickle cell disease (SCD) are at risk of liver injury because of sickle cell hepatopathy and iron overload from chronic transfusions (CT). The authors examine the association between iron overload and liver stiffness measurement (LSM) by vibration controlled transient elastography (VCTE), which has been shown to correlate with fibrosis.

Curative options for sickle cell disease: haploidentical stem cell transplantation or gene therapy?

Haematopoietic stem cell transplantation (HSCT) is curative in sickle cell disease (SCD); however, the lack of available matched donors makes this therapy out of reach for the majority of patients with SCD. Alternative donor sources such as haploidentical HSCT expand the donor pool to nearly all patients with SCD, with recent data showing high overall survival, limited toxicities, and effective reduction in acute and chronic graft-versus-host disease (GVHD). Simultaneously, multiple gene therapy strategies ...


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