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In his keynote speech at a University of Miami symposium recently, Robert M. Califf, the U.S. Food and Drug Administration (FDA) commissioner, marveled about how scientists soon may be able to simply replace disease-causing genes with healthy ones. Gene editing may transform the lives of patients affected with several diseases, including…

High levels of the protein albumin, a condition known as albuminuria and highly prevalent in sickle cell disease (SCD) patients, has now been linked to dysfunctions in cells that line the blood vessels (known as endothelial cells) in SCD patients. The study, “Albuminuria Is Associated with Endothelial Dysfunction and Elevated…

A Phase 2 clinical trial, aiming to assess the effects of hydroxyurea-based therapy at the maximum tolerated dose to prevent stroke in children with sickle cell anemia (SCA), began recently, and the outcomes are expected to highly impact children worldwide, particularly those living in developing countries where the disease is…

Researchers have developed a new protein that can be controlled by a drug and allow the induction of controlled, specific DNA alterations in living cells, without unwanted side-effects. Such developments are a step forward in personalized medicine, especially in diseases with few therapeutic options, as is the case with sickle cell anemia…

Engineers and computer scientists from the University of Washington (UW) have developed what they are calling a HemaApp, designed to detect hemoglobin concentration using simply a smartphone camera and a little extra lighting — rather than needles or an expensive, specialized machine. Measuring hemoglobin, a protein found in red blood cells,…

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…

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…