Health News

A decade after gene therapy, children born with deadly immune disorder remain healthy

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 […]

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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 […]

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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 […]

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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 […]