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Making gene editing accessible in resource limited environments: recommendations to guide a first-time user.
Front Genome Ed
September 2024
Bioengineering and Integrated Genomics Group, Future Production Chemicals Cluster, Council for Scientific and Industrial Research, Pretoria, South Africa.
Article Synopsis
- The CRISPR-Cas9 system has revolutionized genome engineering due to its ease of use, leading to a Nobel Prize win in Chemistry in 2020 for its developers.
- *Recent advancements in gene therapy, particularly in the Global North, showcase its potential benefits in treating diseases like sickle cell anaemia and improving transplant options.
- *However, challenges in the Global South, such as funding limitations and a shortage of skilled professionals, hinder the adoption of this technology, emphasizing the need for cost-effective methods and increased accessibility.*
Successful Kidney Transplant from Donors with Sickle Cell Disease: A Case Series of Six Transplants.
Kidney Med
September 2024
Division of Transplant and Hepatobiliary Surgery, University of Mississippi Medical Center, Jackson, MS.
People with sickle cell disease experience a high incidence of chronic kidney disease and end-stage kidney disease, secondary to tubular and glomerular effects of vaso-occlusion-induced hypoxia. Because of concerns of suboptimal kidney function, sickle cell donors are usually not considered for kidney donation, even if the rest of the parameters are acceptable for organ donation. A significant gap exists between the number of organ donors and the number of candidates waiting for a kidney transplant in the United States.
View Article and Find Full Text PDFAnemia is commonly observed in patients with end-stage renal disease (ESRD) on maintenance hemodialysis (HD) and can be quite severe, particularly when there is an additional comorbidity. With the use of erythropoietin-stimulating agents (ESAs), anemia is effectively treated, but the complete normalization of hemoglobin is not recommended since these agents increase the risk of thrombosis. With improvements in the therapy of sickle cell disease (SCD), patients now survive longer and may more frequently reach end-stage renal disease and require renal replacement therapy.
View Article and Find Full Text PDFNephrological Complications in Hemoglobinopathies: SITE Good Practice.
J Clin Med
December 2023
Dipartimento di Medicina, Università degli Studi di Verona-Azienda Ospedeliera Verona, 37124 Verona, Italy.
Background: Hemoglobinopathies, among which thalassemic syndromes (transfusion-dependent and non-transfusion dependent thalassemias) and sickle cell disease (SCD), are the most widespread monogenic diseases worldwide. Hemoglobinopathies are endemic and spread-out all-over Italy, as result of internal and external migration flows. Nowadays, the increase therapeutic options associated to the general aging of patients with hemoglobinopathies related to the improvement in clinical management, contribute to the abnormalities in kidney function going from blood and urine test alterations to chronic kidney disease and end stage renal disease.
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