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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Storing your child’s cord blood is an incredible way to invest in their future health and well-being. What you might not know: The same cord blood can be used to cure devastating diseases in your other children, or even in you.  Click here to read the story of Hamad, a young Emirati boy who was cured of sickle cell anemia. He wasn’t cured with his own cord blood; he was cured with cord blood stem cells from his younger brother. Cord blood isn’t just an investment in your child; it’s an investment in your family. Call us to learn more about cord blood and cord blood tissue banking. The post Cord...
Source: Cord Blood News - Category: Perinatology & Neonatology Authors: Tags: Cord Blood Cord Tissue parents Source Type: blogs
Disease severity of sickle cell anemia is highly variable, and it is commonly accepted that fetal hemoglobin (HbF) levels play a major role as an ameliorating factor. Investigation of genetic variants have identified several genes to be the principal influencers of HbF regulation. Here, we further elucidated the association of rs4527238 and rs35685045 ofANTXR1 genes in the context of HbF level variance in sickle cell anemia patients of the Arab-Indian haplotype. Samples from 630 sickle cell anemia patients were analyzed for the mutations at 2 specific locations of theANTXR1 gene by TaqMan ®-based real-time PCR. The CC ...
Source: Acta Haematologica - Category: Hematology Source Type: research
In 2017, the Food and Drug Administration approved 2 medications for sickle cell anemia (SCA): hydroxyurea for children and l-glutamine for children and adults. The approval of hydroxyurea was long overdue, but the approval of l-glutamine was a surprise to many. Any effective new treatment for SCA is a welcome advance, but there are few published studies of l-glutamine as a specific treatment for SCA. Accordingly, there are many unanswered questions about its efficacy, safety, and role in current therapy.
Source: Blood - Category: Hematology Authors: Tags: Sickle Cell Disease, Red Cells, Iron, and Erythropoiesis, Blood Spotlight, Clinical Trials and Observations Source Type: research
Abstract Disease severity of sickle cell anemia is highly variable, and it is commonly accepted that fetal hemoglobin (HbF) levels play a major role as an ameliorating factor. Investigation of genetic variants have identified several genes to be the principal influencers of HbF regulation. Here, we further elucidated the association of rs4527238 and rs35685045 of ANTXR1 genes in the context of HbF level variance in sickle cell anemia patients of the Arab-Indian haplotype. Samples from 630 sickle cell anemia patients were analyzed for the mutations at 2 specific locations of the ANTXR1 gene by TaqMan®-based rea...
Source: Acta Haematologica - Category: Hematology Authors: Tags: Acta Haematol Source Type: research
Speaking from the 23rd congress of the European Hematology Association (EHA) 2018 press briefing, held in Stockholm, Sweden, Dr Francesca Vinchi, of the Iron Research Program, New York Blood Center, N... Author: VJHemOnc Added: 08/15/2018
Source: Oncology Tube - Category: Cancer & Oncology Source Type: podcasts
High blood cholesterol is associated with atherogenesis and endothelial dysfunction. The latter is present in hemolytic diseases, such as sickle cell anemia, whose carriers have hypocholesterolemia and low incidence of coronary artery disease.
Source: Journal of Clinical Lipidology - Category: Lipidology Authors: Source Type: research
AbstractPurpose of the ReviewTo provide a clinically useful literature review on the rheumatic manifestations of haemoglobinopathies, critically analysing the literature from the past 5  years.Recent FindingsThere are limited new data to guide the management of rheumatic manifestations of haemoglobinopathies. Data further confirm the wide spectrum of potential rheumatic/MSK involvement in haemoglobinopathies, which poses both a diagnostic and therapeutic challenge. Inflammatory arthritis may be more common than previously believed. Steroid therapy by any route of administration can provocate a potential life-threateni...
Source: Current Rheumatology Reports - Category: Rheumatology Source Type: research
Conclusion: Polymorphisms in the rs7557939 region of the BCL11A gene appear to somehow interfere in the clinical setting of patients with SCD, suggesting relation with the concentration of MetHb and LDH. This study pioneered an investigation into the association of hemolysis biomarkers with BCL11A gene polymorphisms in SCD.RESUMO Introdu ção: Pacientes com anemia falciforme (AF) apresentam hemólise crônica com biomarcadores séricos aumentados. Os polimorfismos genéticos do gene BLC11A modulam a hemoglobina fetal (HbF), reduzindo, assim, a hemólise. Objetivo: Associar os polim...
Source: Jornal Brasileiro de Patologia e Medicina Laboratorial - Category: Pathology Source Type: research
Abstract Sickle cell anemia, a genetic disease caused by a mutation in the beta-globin gene, can present oral manifestations such as delayed tooth eruption and hypomineralized enamel and dentin. The aim of the present study was to evaluate the prevalence and severity of developmental defects of enamel (DDE) and delayed tooth eruption in children with sickle cell anemia. The sample comprised 56 male and female children with sickle cell anemia aged 6 to 12 years and treated at the Hematology and Hemotherapy Center of Pernambuco, Brazil. The data were collected according to the WHO criteria for DDE and tooth eruption. The pre...
Source: Brazilian Oral Research - Category: Dentistry Source Type: research
Introduction: Hydroxyurea is an antimetabolite used to treat myeloproliferative disorders, solid tumors, and sickle cell anemia, which has been well documented to cause lower extremity ulcers. Discontinuing the medication may result in spontaneous ulcer resolution. However, hydroxyurea-induced skin ulcers often heal slowly over months to years, causing considerable morbidity.
Source: Journal of the American Academy of Dermatology - Category: Dermatology Source Type: research
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