Tag: <span>Sickle Cell Disease</span>

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Corticosteroids raise the risk of hospitalization for pain crises among individuals living with sickle cell disease

AMERICAN SOCIETY OF HEMATOLOGY (WASHINGTON, April 26, 2022) — People with sickle cell disease (SCD) who were recently prescribed a corticosteroid – a medicine frequently used to treat asthma or inflammation – were found to be significantly more likely to be hospitalized for a severe pain event, according to a paper published today in the journal Blood. The...

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Experimental Gene Therapy Reverses Sickle Cell Disease for Years

A study of an investigational gene therapy for sickle cell disease has found that a single dose restored blood cells to their normal shape and eliminated the most serious complication of the disease for at least three years in some patients. Four patients at Columbia University Irving Medical Center/NewYork-Presbyterian participated in the multicenter study, the first to...

New insight into red blood cell machinery offers clues to treating sickle cell disease
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New insight into red blood cell machinery offers clues to treating sickle cell disease

by St. Jude Children’s Research Hospital Corresponding author Mitchell Weiss, M.D., Ph.D., chair of the St. Jude Department of Hematology (left), helped uncover how the body generates protective fetal hemoglobin against sickle cell disease and beta-thalassemia. Credit: St. Jude Children’s Research Hospital Hematologists at St. Jude Children’s Research Hospital have discovered key molecular details of...

Treatment Options for Sickle Cell Disease
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Treatment Options for Sickle Cell Disease

By Dr. Osman Shabir, Ph.D. Reviewed by Emily Henderson, B.Sc. Routine treatments for sickle cell disease (SCD) are currently limited to blood, stem cell, and bone marrow transplants through other treatment and management strategies are available to help manage anemia and painful episodes amongst other complications with even more promising drugs and treatments being developed...

Toward a reliable oral treatment for sickle cell disease
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Toward a reliable oral treatment for sickle cell disease

by  American Chemical Society This X-ray structure shows FTX-6058 bound to its protein target. Credit: Fulcrum Therapeutics For the millions of people worldwide who have sickle cell disease, there are only a few treatment options, which include risky bone marrow transplants, gene therapy or other treatments that address a subset of symptoms. Today, researchers will describe the discovery...

UC Consortium Launches First Clinical Trial Using CRISPR to Correct Gene Defect That Causes Sickle Cell Disease
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UC Consortium Launches First Clinical Trial Using CRISPR to Correct Gene Defect That Causes Sickle Cell Disease

Scientists at UC San Francisco, UC Berkeley and UCLA have received U.S. Food and Drug Administration approval to jointly launch an early phase, first-in-human clinical trial of a CRISPR gene correction therapy in patients with sickle cell disease using the patient’s own blood-forming stem cells. The trial will combine CRISPR technology developed at Innovative Genomics Institute (IGI)...

NIH scientists discover how DNA fragments can trigger inflammation in sickle cell disease
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NIH scientists discover how DNA fragments can trigger inflammation in sickle cell disease

Researchers have discovered that DNA from the mitochondria – the cell’s “powerhouses” – acts as a danger signal in the body and triggers inflammation in people with sickle cell disease. A better understanding of mitochondrial DNA, long known to circulate in human blood, may provide vital insight into how to stop the underlying chronic inflammation that...

Gene therapy gives man with sickle cell disease the chance for a better future
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Gene therapy gives man with sickle cell disease the chance for a better future

UNIVERSITY OF CALIFORNIA – LOS ANGELES HEALTH SCIENCES IMAGE: EVIE JUNIOR CREDIT: UCLA BROAD STEM CELL RESEARCH CENTER For Evie Junior, living with sickle cell disease has been like running a marathon. “But it’s a marathon where as you keep going, the trail gets rockier and then you lose your shoes,” the 27-year-old said. “It gets harder...

New technology diagnoses sickle cell disease in record time
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New technology diagnoses sickle cell disease in record time

UNIVERSITY OF COLORADO AT BOULDER IMAGE: AN ACOUSTO THERMAL SHIFT ASSAY “LAB-ON-A-CHIP ” DEVICE SHOWN NEXT TO A QUARTER FOR SIZE COMPARISON. Researchers at the University of Colorado Boulder and the University of Colorado Anschutz Medical Campus have developed a new way to diagnose diseases of the blood like sickle cell disease with sensitivity and...