For most people with hemophilia B, whose bodies can’t properly form blood clots, constant injections to replenish their clotting factors are a way of life. But now, Salk researchers have demonstrated in mice that hemophilia B can be treated for life with one single injection, containing disease-free liver cells that can produce their missing clotting
Biomedical engineers at Duke University have used a CRISPR/Cas9 genetic engineering technique to turn off a gene that regulates cholesterol levels in adult mice, leading to reduced blood cholesterol levels and gene repression lasting for six months after a single treatment. This marks the first time researchers have delivered CRISPR/Cas9 repressors for targeted therapeutic gene
Boston University researchers, Thomas Kepler, professor of microbiology; Stephanie Pavlovich, an MD/PhD student; and Elke Mühlberger, director, Biomolecule Production Core, National Emerging Infectious Diseases Laboratories (NEIDL); in collaboration with the United States Army Medical Research Institute of Infectious Diseases (USAMRIID) and the Centers for Disease Control and Prevention’s (CDC) Viral Special Pathogens Branch, today released
Doctors at the University of Illinois Hospital have cured seven adult patients of sickle cell disease, an inherited blood disorder primarily affecting the black community, using stem cells from donors previously thought to be incompatible, thanks to a new transplant treatment protocol. The doctors report on the new technique in the journal Biology of Blood
Donor age does not appear to influence the functionality of stem cells derived from adult body tissues, concludes a new review. The analysis of research on induced pluripotent stem cells (iPSCs) finds that not only are typical signs of aging reversed in iPSCs, but cells derived from both older and younger donors show the same
New research indicates that belimumab, a monoclonal antibody therapy that targets a component of the immune system, provides considerable benefits to patients with systemic lupus erythematosus (SLE), a predominately female, chronic inflammatory disease that can affect virtually any organ. The Arthritis & Rheumatology findings are encouraging because lupus is among the leading causes of death
Chronic lymphocytic leukemia (CLL) is a haematological malignancy that originates in the blood-forming cells of the bone marrow and spreads to other organs through the bloodstream. When infiltrating tissues, CLL cells come in contact with healthy cells, including immune cells. To ensure their survival and growth, CLL cells are able to establish a microenvironment in
A team from the Spanish National Cancer Research Centre (CNIO) has determined for the first time the high-resolution structure of a complex (R2TP) involved in key processes for cell survival and in diseases such as cancer. This achievement has been made possible by using high-resolution cryo-electron microscopy, a technique brought to the CNIO thanks to
Researchers at the University of Sydney have established a method to identify individual nanoparticles released by human cells, opening the way for them to become diagnostic tools in the early-detection of cancers, dementia and kidney disease. The particles, known as extracellular vesicles, or EVs, are routinely released by cells and play a central role in
Atherosclerosis is the most common cause of death and disease in the Western world. In Germany, about 300,000 people each year suffer a heart attack and some 270,000 a stroke as a result of the condition. Atherosclerosis is estimated to be responsible for a little more than half of all deaths in these countries. Searching
Invariant natural killer T (iNKT) cells are powerful weapons our body’s immune systems count on to fight infection and combat diseases like cancer, multiple sclerosis, and lupus. Finding ways to spark these potent cells into action could lead to more effective cancer treatments and vaccines. While several chemical compounds have shown promise stimulating iNKT cells
One of the major side effects of chemotherapy is a sharp drop in white blood cells, which leaves patients vulnerable to dangerous infections. MIT researchers have now developed a portable device that could be used to monitor patients’ white blood cell levels at home, without taking blood samples. Such a device could prevent thousands of
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