PHILADELPHIA – Data presented at the American Academy of Neurology (AAN) meeting this week on experimental therapeutics for Huntington's disease gave some cause for optimism. And, as good research does, they identified new questions as they answered current ones.
Researchers have linked the hormone oxytocin, which stimulates social and pair bonding, to the increased risk of aortic tear in women with Marfan syndrome.
"The molecular genetics for APP" – amyloid precursor protein – "still unequivocally demonstrate that APP and Abeta are crucial for disease pathogenesis" in Alzheimer's disease (AD), Carlo Condello told BioWorld.
Researchers have discovered that the synaptic protein Bassoon accumulated in the neurons of mice with experimental autoimmune encephalitis, the closest animal model to multiple sclerosis (MS), causing neuronal damage in much the same way that protein aggregates damage neurons in neurodegenerative diseases.
Three phase I trials – I-PREDICT, TARGET and WINTHER – published back to back in the April 22, 2019, issue of Nature Medicine have reported progress on a central goal of precision medicine: the rapid delivery of therapy tailored to the molecular details of a patient's illness.
Researchers from Memorial Sloan-Kettering Cancer Center have identified the secondary bile acid lithocholic acid (LCA) as a contributor to a switch from individual growth to the formation of bacterial chains by the hospital-acquired pathogen vancomycin-resistant enterococci (VRE), and have shown that the formation of such chains facilitated biofilm formation and increased antibiotic resistance.
Researchers from Albert Einstein Medical College and the University of Pittsburgh Medical Center have identified brain protective functions of recombinant tissue plasminogen activator (tPA) beyond its main effect of breaking down blood clots, and a compound that could target the same functions without affecting clotting.
Well, this sounds familiar: Researchers have identified a member of the sirtuin family that affected longevity. Across 18 different rodent species, those with more effective double-stranded break DNA repair – but not in nucleotide excision repair – by Sirtuin 6 (SIRT6) also had longer life spans.
Several infants treated with autologous ex vivo gene therapy for newly diagnosed X-linked severe combined immunodeficiency (X-SCID) in a phase I/II trial have developed fully functioning immune systems, including T cells, B cells and natural killer cells. The results, which were published in the April 17, 2019, issue of The New England Journal of Medicine, mark the first time that first-line treatment with X-SCID gene therapy has resulted in an immune system that was able to generate not just T cells, but the major cell types of the innate and adaptive immune systems.
Researchers at the University of Texas at Dallas have gained new insight into sex differences in response to calcitonin gene-related peptide (CGRP) that may underlie the higher prevalence of migraine in females.