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A Possible Novel Therapy for a Rare but Potentially Fatal Blood Disorder

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A transgenic mouse model is a proof-of-concept that platelet blood cells that are loaded with the enzyme ADAMTS13 can be an effective treatment in murine models of thrombotic thrombocytopenic purpura.

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Research Reveals High Prevalence of Sleep Disordered Breathing in Adults with Sickle Cell Disease

Adults with sickle cell disease who report trouble with sleep could actually have a clinical diagnosis of sleep disordered breathing which could lower their oxygen levels at night.

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TSRI Team Discovers Enzyme that Keeps Blood Stem Cells Functional to Prevent Anemia

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Scientists at The Scripps Research Institute have found a particular enzyme in blood stem cells that is key to maintaining healthy periods of cell inactivity; if these cells become too active and divide too often, they risk acquiring cell damage and mutations.

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Study Bolsters "Turbocharged" Protein as a Promising Tool in Hemophilia Gene Therapy

Using gene therapy to produce a mutant human protein with unusually high blood-clotting power, scientists successfully treated dogs with the bleeding disorder hemophlia, without triggering an unwanted immune response.

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New Gene Therapy for Hemophilia Shows Potential as Safe Treatment

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New Research from San Raffaele Telethon Institute for Gene Therapy and the University of North Carolina showed that a reprogrammed retrovirus could successfully transfer new clotting genes into animals with hemophilia B to safely and dramatically decrease spontaneous bleeding.

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Johns Hopkins Researchers Engineer Custom Blood Cells

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Researchers at Johns Hopkins have successfully corrected a genetic error in stem cells from patients with sickle cell disease, and then used those cells to grow mature red blood cells, they report. The study represents an important step toward more effectively treating certain patients with sickle cell disease who need frequent blood transfusions and currently have few options.

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Investigational Therapy Could Attack Cause of Sickle Cell Crises

Treatment for painful episodes of blood vessel obstruction in sickle cell anemia is currently limited to controlling pain, but an investigational therapy might be able to interfere with the underlying cause of these events, known as vaso-occlusion crises, researchers at Duke Medicine report.

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Quick Antibiotics Reduce PICU Needs and Mortality of Pediatric Cancer Patients

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A University of Colorado Cancer Center study published in the journal Pediatric Blood & Cancer shows that pediatric cancer patients who receive antibiotics within 60 minutes of reporting fever and showing neutropenia (low neutrophil count), go on to have decreased intensive care needs and lower mortality compared with patients who receive antibiotics outside the 60-minute window.

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The Immune System May Play a Key Role in Viral Therapy’s Effectiveness Against Tumors

Viral therapy for childhood cancer could possibly improve if treatments such as chemotherapy do not first suppress patients’ immune systems, according to findings published today in the journal Molecular Therapy—Oncolytics.

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Platelet Transfusions Increase Death Rate in Some Blood Cell Disorders

People hospitalized with certain rare blood cell disorders frequently receive a treatment that is associated with a two- to fivefold increase in death, according to a new study that reviewed hospital records nationwide. The study authors recommend that for these rare disorders, doctors should administer the treatment, a platelet transfusion, only in exceptional circumstances.