A new engineered gene therapy virus, inserted into blood stem cells and then transplanted into mice with sickle cell disease, markedly reduced red blood cell damage according to the study “Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype,” published in the Journal of Clinical Investigation. A clinical gene therapy trial…
News
A recent study suggests that sickle cell anemia (SCA) can be detected by a test using samples collected from inside the mouth of patients. This test allows for the detection of micronucleus, a deficient-looking cell nucleus that shows up in cells from a defective process of cell multiplication. The study,…
Sickle cell disease (SCD) is rampant in sub-Saharan Africa, a region that accounts for about two-thirds of all SCD births worldwide, and among the disease’s complications can be an imbalance in elements such as iron, an essential trace element for good health. Researchers compared key proteins associated with iron metabolism between children with SCD and…
Researchers believe that activating a specific oxidative stress regulator with sulforaphane (SFN), a chemical compound found in vegetables such as broccoli and brussels sprouts, might have a therapeutic benefit in patients with sickle cell disease (SCD). They tested their hypothesis in a Phase 1 study that evaluated broccoli sprout homogenate (BSH), which naturally contains SFN,…
The Embrace Kids Foundation has committed $70,000 to support the expansion of Rutgers Robert Wood Johnson Medical School (RWJMS) Comprehensive Sickle Cell Center at Rutgers Cancer Institute of New Jersey (CINJ) to include a pediatric sickle cell and hemoglobinopathies nurse navigator position. The main focus of the new position is…
The American Red Cross and Sports Clips Haircuts have partnered to increase blood and platelet donations across the U.S., offering a coupon for a free haircut to all those who donate during September. Blood donations can be life-saving for people with chronic conditions like sickle cell disease (SCD). “The Red Cross and Sport…
Emmaus Life Sciences announced that expects to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) next month for its lead investigational treatment for sickle cell disease. Emmaus’ lead candidate is an oral pharmaceutical grade L-glutamine treatment that demonstrated positive clinical results in…
The William E. Proudford Sickle Cell Fund in celebration of September’s National Sickle Cell Awareness Month is recognizing the accomplishments of Dr. Gary Gibbons, Director of the National Heart, Lung, and Blood Institute (NHLBI). “Under the leadership of Dr. Gibbons, NHLBI has demonstrated…
In sickle cell anemia (SCA), infection with the erythrovirus B19 (B19V) is known to cause transient aplastic crises, in which production of red blood cells (RBCs) in the bone marrow fails. In this study, researchers describe the clinical course and lab tests of two brothers with sickle cell disease who had the infection.
Researchers developed a mobile app to provide primary care physicians (PCPs) with tools — guidelines, algorithms, and a pathway — needed to communicate effectively with local specialists regarding sickle cell disease. The team from Duke University and the University of Pittsburgh developed the iOS-based ‘toolkit’ after finding that not all…
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