A multitude of genes have been linked to the development of Alzheimer's disease. Specifically how those genes might influence the progression of neurodegeneration remains something of a black box though, in part because of the challenges of examining in molecular detail the brain of a living patient. Using cerebrospinal fluid (CSF) collected from living patients, a team of researchers at Washington University School of Medicine in St. Louis has for the first time linked disease-related proteins and genes to identify specific cellular pathways responsible for Alzheimer's genesis and progression.

The U.S. Food and Drug Administration approved Kebilidi (eladocagene exuparvovec-tneq), an adeno-associated virus vector-based gene therapy indicated for the treatment of adult and pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency. Kebilidi is the first FDA-approved gene therapy for treatment of AADC deficiency.

Scientists at the University of Copenhagen have discovered a new weight loss drug target that reduces appetite, increases energy expenditure, and improves insulin sensitivity without causing nausea or loss of muscle mass. The discovery was reported in the journal Nature and could lead to a new therapy for millions of people with both obesity and type 2 diabetes who do not respond well to current treatments. Millions of people around the world benefit from weight loss drugs based on the incretin hormone GLP-1.

Immune cells are capable of detecting infections just like a sniffer dog, using special sensors known as Toll-like receptors, or TLRs for short. But what signals activate TLRs, and what is the relationship between the scale and nature of this activation and the substance being detected? In a recent study, researchers from the University of Bonn and the University Hospital Bonn (UKB) used an innovative method to answer these questions. The approach that they took might help to speed up the search for drugs to combat infectious diseases, cancer, diabetes or dementia.

Researchers at Uppsala University and KTH Royal Institute of Technology have developed a new form of precision medicine, an antibody, with the potential to treat several types of cancer. Researchers have managed to combine three different functions in the antibody, which together strongly amplify the effect of T cells on the cancer tumour. The study has been published in Nature Communications. Researchers have developed a unique type of antibody that both targets and delivers a drug package via the antibody itself, while simultaneously activating the immune system ("3-in-1 design") for personalised immunotherapy treatments.