News

For the first time, scientists were able to correct the genetic mutation that causes sickle cell disease in stem cells. In a collaborative effort, researchers at UC Berkeley, UC San Francisco Benioff Children’s Hospital Oakland Research Institute (CHORI), and the University of Utah School of Medicine fixed the mutation…

A published case study reports that patients with mildly symptomatic sickle cell disease (SCD) can exceed the U.S. median life expectancy  of 47 years for patients with the disease if it is managed properly. The report published in Blood, the Journal of the American Society of…

Researchers at various institutions from the African country of Cameroon recently found a link between oxidative stress and sickle cell disease (SCD). The study, “Oxidative profile of sickle cell patients in a Cameroonian urban hospital,” was published in…

Mast Therpeutics has announced that the clinical development of Vepoloxamer (MST-188) is likely to be terminated. Results from a Phase 3 clinical trial evaluating the investigational drug in patients with sickle cell disease (SCD) failed to meet its primary goal. Brian M. Culley, Mast Therapeutics’ chief executive…

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…