Over a decade ago, UCLA physician-scientists began using a pioneering gene therapy they developed to treat children born with a rare and deadly immune system disorder. They now report that the effects of the therapy appear to be long-lasting, with 90% of patients who received the treatment eight to 11 years ago still disease-free. ADA-SCID, or adenosine deaminase–deficient […]
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New gene therapy uses Tylenol to combat genetic diseases
Researchers have developed a new approach to gene therapy that leans on the common pain reliever acetaminophen to force a variety of genetic diseases into remission. A paper published in Science Translational Medicine describes how the novel technique successfully treated the blood-clotting disorder hemophilia and the debilitating metabolic disease known as phenylketonuria, or PKU, in […]
Discovery of potential new combination therapy for leukemia
WEHI researchers have discovered why some leukemia cells are resistant to new “SMAC-mimetic’ anti-cancer agents, a finding which could improve the use of these drugs in the clinic. The team discovered that the ‘drug pump’ protein MDR1 makes acute myeloid leukemia (AML) cells resistant to SMAC-mimetics, by rapidly pumping out the drug of the cells […]
New potential therapy for Crohn’s disease in children
Scientists from the Stanley Manne Children’s Research Institute at Ann & Robert H. Lurie Children’s Hospital of Chicago demonstrated that a nanotherapy reduces intestinal inflammation and shrinks lesions in a rodent model of severe Crohn’s disease. This approach could become an alternative to biologic antibody therapies that carry many side effects, including increased risk of […]
Researchers use mathematical modeling to identify factors that determine adaptive therapy success
One of the most challenging issues in cancer therapy is the development of drug resistance and subsequent disease progression. In a new article featured on this month’s cover of Cancer Research, Moffitt Cancer Center researchers, in collaboration with Oxford University, report results from their study using mathematical modeling to show that cell turnover impacts drug […]
Response to infection therapy better understood thanks to a new technique
Infectious diseases are caused by pathogenic viruses, bacteria, fungi or parasites. The treatment of bacterial and fungal infections relies particularly on antimicrobial drugs, while the focus in treating viral infections is the alleviation of symptoms. Initial therapy for infection is often empiric and guided by clinical presentation. Its efficacy on the pathogen is, however, only […]
US approves COVID therapy, G20 pushes for global vaccine access
US drug regulators gave emergency approval to a COVID-19 antibody therapy on Saturday and G20 nations pushed for global access to vaccines as the pandemic led to further closures in parts of the world. With cases surpassing 12 million in the United States, the highest in the world, many Americans were nonetheless heading to airports […]
Experimental hormone therapy may speed recovery for COVID patients
Every time the phone rang, it seemed to Kristine Smoley like more bad news about her husband, who contracted COVID-19 and was in a hospital intensive care unit on a ventilator because he couldn’t breathe on his own. Smoley was prepared for the worst when a nurse from Duluth’s Essentia Health called with hope—albeit with […]
What’s up doc? Donkey therapy eases Spain medics’ stress
Walking into a dusty paddock, a young nurse is quickly surrounded by a group of donkeys gently nudging her for attention as she strokes their soft noses and feeds them carrots. When someone places a 10-day-old colt in her arms, Monica Morales squeals with delight, visibly relaxed after a few hours unwinding at El Burrito […]
How immune cells can recognize—and control—HIV when therapy is interrupted
Immune cells that can recognize residual HIV-infected cells in people living with HIV (PLWH) who take antiretroviral therapy (ART) remain active for years, says a new study published today in eLife. The findings also suggest the majority of these immune cells, called CD8+ T cells, should have the capacity to detect the HIV-infected cells that […]