Category: Drugs development & discovery
- Ivacaftor reduces experimental lung fibrosis and senescence in preclinical models ()
Published November 3, 2026. Ivacaftor (VX-770), a CFTR-potentiating drug, reduced lung scarring and cellular senescence in experimental models reported in Cell Death Discovery on October 31. In mice with bleomycin-induced pulmonary fibrosis, treatment reduced collagen deposition and improved lung structure and function. It also reduced abnormal KRT8-positive transitional cells and preserved alveolar type 1 cells. […]
- Dent disease gene therapy: lasting expression does not guarantee kidney benefit in mice ()
Published November 3, 2026. A mouse study of Dent disease type 1 shows why persistent gene expression alone is an incomplete measure of gene-therapy success. Researchers at Wake Forest compared renal delivery of human CLCN5 using lentiviral and AAV9 vectors. In mutant adult mice, combining lentiviral delivery with locally administered rapamycin-loaded biodegradable microspheres improved ClC-5 […]
- Cardiac gene therapy review highlights delivery routes and biomaterial trade-offs ()
Published November 2, 2026. A review published today in npj Biomedical Innovations examines a practical bottleneck in cardiac gene therapy: getting enough therapeutic vector into the heart without excessive exposure elsewhere. Kabboul and colleagues compare established and experimental administration routes for adeno-associated virus vectors. Rapid coronary blood flow, endothelial barriers, limited retention and immune responses […]
- Prolactin receptor splice modulation shows preclinical promise in lupus ()
Published November 2, 2026. A study published today in Experimental & Molecular Medicine investigates alternative forms of the prolactin receptor as a potential target in systemic lupus erythematosus. Rodrigues Lima-Junior and colleagues found an increased balance of long versus short receptor isoforms in lupus immune cells. A splice-modulating oligomer reduced the long form while increasing […]
- Insulin improves experimental stem-cell and vesicle effects in diabetic mice ()
Published November 2, 2026. A Cell Death & Disease study published today explores why insulin can improve the durability of experimental mesenchymal stem-cell treatment in mouse models of type 2 diabetes. Hu and colleagues tested umbilical cord-derived mesenchymal stem cells in db/db mice and diet-induced obese mice. Cells alone produced short-term metabolic improvements, whereas combining […]
- DeepDelivery AI identifies human-derived protein scaffolds for experimental mRNA delivery ()
Published November 1, 2026. A Nature Communications study published on October 31 introduces DeepDelivery, an AI platform that searches human proteins for components capable of supporting engineered mRNA delivery. Xun and colleagues screened the human proteome computationally and nominated 512 candidates. Of 15 selected proteins tested experimentally, 12 supported particle formation and mRNA delivery within […]
- KIF2C activation improves DNA repair and extends lifespan in progeria mice ()
Published November 1, 2026. A study published today in Nature Communications investigates whether activating the kinesin protein KIF2C can improve DNA repair and reduce features of premature aging. Macedo and colleagues tested a small-molecule KIF2C agonist in aged cells and cells derived from people with Hutchinson–Gilford progeria syndrome or Down syndrome. They report improved repair […]
- ROBO1-targeted CAR T cells show preclinical activity against pediatric solid tumors ()
Published November 1, 2026. A study published in Nature Communications on October 31 identifies ROBO1 as a candidate CAR T-cell target for several pediatric solid tumors. Buhl and colleagues compared cell-surface proteins in patient-derived malignant rhabdoid tumor organoids and matched normal kidney organoids, integrating these measurements with gene-expression and protein data. ROBO1 showed strong, uniform […]
- Glioblastoma target GD2 changes with TGF-beta signaling in two cell-line models ()
Published October 31, 2026. GD2, a potential therapeutic target in glioblastoma, can change in opposite directions depending on the tumor cell model and its signaling environment. A study published today in Scientific Reports examined this variability in two laboratory cell lines. Researchers exposed U87-MG and U251-MG cells to TGF-β and measured gangliosides, gene expression, surface […]
- Research highlight examines age-specific targets for anti-tumor T cell function ()
Published October 31, 2026. An October 29 research highlight in Cell Research examines why age may matter when selecting targets to improve anti-tumor T cell function. It discusses an earlier Cell study of Dusp5 and Zfp219, rather than reporting a new clinical trial. In the original experiments, single-cell CRISPR screens identified two different routes to […]