CAMBRIDGE, England – Researchers have discovered that misreading of therapeutic mRNAs by the cell’s decoding machinery can cause an unintended immune response in the body. They have identified the sequence within the mRNA that causes this to occur and found a way to prevent ‘off-target’ immune responses to enable the...
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BALTIMORE, MD — Researchers at Kennedy Krieger Institute and Boston Children’s Hospital have discovered a new way to decrease early seizure likelihood and improve neurologic outcomes among infants with Sturge-Weber Syndrome (SWS). This collaboration, funded by National Institutes of Health, is the first multi-center study of its kind focused on...
ANN ARBOR, Michigan — To understand why some cancers successfully circumvent the immune system to grow unchecked, researchers turned to pregnancy. “In pregnancy, the immune system does not reject the growing fetus, so we know there must be mechanisms active in the placenta. In cancer, it’s the same thing: the...
Houston, Texas – An international team of researchers has provided a genetic diagnosis for 30 individuals whose condition was undiagnosed for years despite extensive clinical or genetic testing. The study, conducted by researchers at Baylor College of Medicine, National University of Singapore and collaborating institutions worldwide, appeared in Genetics in...
Sidney, Australia – Innovative study of DNA’s hidden structures may open up new approaches for treatment and diagnosis of diseases, including cancer. DNA is well-known for its double helix shape. But the human genome also contains more than 50,000 unusual ‘knot’-like DNA structures called i-motifs, researchers at the Garvan Institute...
Troy, New York – Using artificial intelligence tools to analyze years of biomedical data, researchers at Rensselaer Polytechnic Institute have discovered a possible connection between sleep, gastrointestinal health, and two potentially harmful behaviors often associated with profound autism: self-injury and aggression. Their study is published in the Journal of Personalized...
Leipzig, Germany – Every year, around 4,000 people in Germany are diagnosed with myelodysplastic neoplasms (MDS). The disease can lead to anaemia, infections and an increased risk of bleeding. People with higher-risk MDS have a significantly lower life expectancy than the average person of the same age. In the higher-risk...
MILAN, Italy – Beta-thalassemia (Bthal) is a genetic disorder due to mutations in the ß-globin gene, leading to a reduced or absent production of HbA, which interferes with erythroid cell maturation and limits red cell production. Patients are affected by severe anemia, hepatosplenomegaly, and skeletal abnormalities due to rapid expansion...
Using data generated from patients and mice with genetic mutation for the disorder Usher syndrome, researchers from the University of Maryland School of Medicine (UMSOM), the National Institutes of Health’s National Eye Institute (NEI), and National Institute on Deafness and Other Communication Disorders (NIDCD), documented the natural history of vision...
Houston, TX – Texas Children’s Hospital, an internationally-recognized, top-ranked children’s hospital and pediatric research center affiliated with Baylor College of Medicine, is the first to deliver a novel gene therapy to treat Rett syndrome in pediatric patients. Two female patients with Rett syndrome were the first children worldwide to receive...