Demystifying Medicine Lecture Series: Sickle Cell Anemia: Treatable and/or Curable?
Date: Tuesday, 04 14, 2020; Speaker: John Tisdale, MD, NHLBI; Alan Schechter, MD, NIDDK; Building: Building 50; 1227/1233; CME Credit; Videocast Event
Date: Tuesday, 03 03, 2020; Speaker: John Tisdale, MD, NHLBI; Alan Schechter, MD, NIDDK; Building: Building 50; 1227/1233; CME Credit; Videocast Event
Conclusion: Endocrinopathy is a frequent complication of hemoglobinopathies, for which the most common were found to be short stature, diabetes mellitus, and low bone mineral density. PMID: 32020146 [PubMed - in process]
The objective of this study is to reduce the morbidity of children with homozygous sickle cell disease presenting severe forms. We have conducted a longitudinal study between November 2015 and September 2017 at the Brazzaville University Hospital. Children with sickle cell disease requiring treatment with hydroxyurea were included. The variables studied were therapeutic compliance, evolutive profile of nutritional status, indications of hydroxyurea, electrophoresis of hemoglobin, blood count, and toxicity. The statistical test used was Student test with a significance threshold of less than 0.05. One thousand twe...
ConclusionIt clear that this study has successfully identified LCR mutations for random Saudi patients with SCD. The above results should be taken further to set up management strategies to improve outcomes.
Thalassemia major (TM) and sickle cell anemia (SCA) are the two most widespread inborn hemoglobinopathies worldwide. TM was the first congenital hemoglobinopathy to be cured by allogenic hematopoietic stem cell transplantation (HSCT),1 and SCA rapidly followed.2,3 Other than gene therapy,4 HSCT remains the only available curative treatment for these two diseases. Provided that an HLA-identical sibling donor is available, it should be performed as soon as possible for TM and in case of complications for SCA.
Contributors : Alexander Azzo ; Xiaofei Yu ; Mikhail Dozmorov ; Gordon D GinderSeries Type : Expression profiling by high throughput sequencingOrganism : Homo sapiensAs high fetal hemoglobin (HbF) levels ameliorate the underlying pathophysiologic defects in sickle cell anemia and β-thalassemia, understanding the mechanisms that enforce silencing of HbF postnatally offers the promise of effective molecular therapy. Depletion of Methyl cytosine Binding Domain protein 2 (MBD2) causes a 10-20 fold increase in γ-globin gene expression in adult β-YAC transgenic mice. To determ ine the effect of MBD2 depletion in ...
(University of Washington Health Sciences/UW Medicine) 3D microvessels have been created to observe how red blood cells transit ultra-small blood vessels. They squeeze single-file through microvessels to bring oxygen and nutrients. Red cells burdened with malaria stall, blocking the blood vessel. The platform is expected to have other uses in studies of how microvascular damage occurs in diabetes and sickle cell anemia. They might be further developed to supply blood circulation to organ repair patches or to 3D printed transplants.
(Georgia Institute of Technology) In diseases like cancer, atherosclerosis, and sickle cell anemia, cathepsins promote their propagation. Drug trials to inhibit these enzymes have failed due to baffling side effects. Now a new study examines cathepsins in systems to remove some of the bafflement.
It is believed that the current prevalence of malaria in endemic areas reflects selection for the carrier form of sickle cell trait through a survival advantage. Malaria has been incriminated as a great cause ...
Conclusions: For most of the ANC services, reason for nonreceipt of the service as given by the HRMs was “service not offered.”