Over a period of nearly 3 decades, gene therapy for the inherited bleeding disorder hemophilia progressed from basic science to approval of 3 gene therapy drugs that deliver coagulation factor genes to hepatocytes using adeno-associated viral vectors. These molecular medicines can provide multi-year therapy after a single drug administration.
Li et al. reveal insights into female meiosis by applying single-cell and low-input sequencing methods to mouse embryonic gonads. They uncover significant variabilities in recombination…
Fermentation products are the most abundant gut microbial metabolites absorbed by the host, with important dose-dependent consequences for health. In this study, we present a…
Lambert et al. describe an antibody-based approach targeting the pleiotropic TGF-β1 cytokine in a manner unexplored thus far. At variance with existing TGF-β1-targeting strategies aiming…
Beyond its established role in glycolysis, PFKFB3 directly phosphorylates CAD to promote de novo pyrimidine biosynthesis. This dual function enables PFKFB3 to regulate cell growth…
Competition between transcription factors and mismatch repair machinery drives localized hypermutation at regulatory elements, with implications for cancer and genome evolution.
Su et al. elucidated the cryo-EM structures of the CASTOR1-GATOR2, Sestrin2-GATOR2, and CASTOR1-Sestrin2-GATOR2 complexes. By integrating the structural findings with in vitro pull-down assays and…
Competition between transcription factors and mismatch repair machinery drives localized hypermutation at regulatory elements, with implications for cancer and genome evolution.
D’Oliveira et al. reported an integrative single-cell multi-omic analysis linking T1D genetic risk to specific pancreatic cell types. They identified β cell-mediated risk at the…
This collection showcases a sampling of research and perspectives from our journals on the use of artificial intelligence in the study, diagnosis, and treatment of…
This collection showcases a sampling of research and perspectives from our journals on the use of artificial intelligence in the study, diagnosis, and treatment of…
