ST. JUDE CHILDREN’S RESEARCH HOSPITAL IMAGE: CORRESPONDING AUTHOR MITCHELL J WEISS, M.D., ST. JUDE DEPARTMENT OF HEMATOLOGY CHAIR; FIRST AUTHOR RUOPENG FENG, PH.D., ST. JUDE DEPARTMENT OF HEMATOLOGY CREDIT: COURTESY OF ST. JUDE CHILDREN’S RESEARCH HOSPITAL (MEMPHIS, Tenn. – October 12, 2022) Scientists at St. Jude Children’s Research Hospital have shown how a protein responsible...
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...
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...
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...
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...
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...
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)...
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...
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...
CONN HASTINGS, OCTOBER 16TH, 2020 Researchers at the University of Colorado have developed a new rapid test for sickle cell disease. Their tiny device is less than the size of a quarter, and can provide a result in as little as one minute. The technology uses ultrasound to heat a protein sample and then measures how...
