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.
Noninvasive electrical stimulation of the brain to synchronize the activity of distant cortical regions could boost the working memory of trial participants in their sixties to resemble a group of individuals in their twenties.
Scientists from Brigham and Women's Hospital have successfully targeted the enzyme myosin light chain kinase 1 (MLCK1) to improve the symptoms of inflammatory bowel disease, while avoiding the toxicity that has doomed previous approaches.
No good deed goes unpunished. Successful development of a PD-1/PD-L1 checkpoint blocker, for example, can get you a grilling by Richard Pazdur, director of the FDA's Oncology Center of Excellence at the annual meeting of the American Association for Cancer Research (AACR).
ATLANTA – CAR T cells are currently capable of making a giant difference, but only to tiny numbers of patients. But at the annual meeting of the American Association for Cancer Research, both phase I trial and novel preclinical approaches were on view that could bring the approach to more indications with larger numbers of patients.
Many hair follicles that stop producing hairs contain quiescent stem cells, suggesting that waking up those cells could reverse hair loss. Researchers at Columbia University have discovered that the hair follicle stem cells are actively restrained by tissue macrophages.
By investigating the "surfaceome," the group of proteins that move to the cell surface in response to KRAS signaling, researchers have identified a protein, Syndecan-1, that is critical for KRAS-driven cancer cells to obtain nutrients from the environment.
