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Gene therapies featured prominently among this year's newsworthy drugs, some of which have already received a green light from regulatory agencies for sale or are otherwise surging forward in trials. Other drugs ended the year with a much less rosy efficacy or safety profile.
Cancer-derived induced pluripotent stem cells provide a new opportunity to model the effects of the cancer genome. In this Perspective, Eirini Papapetrou discusses the future applications of these cells for cancer modeling and therapeutic understanding.
B cells protect against inflammation-associated preterm labor via IL-33-induced PIBF1 expression in mice, which suggests a therapy for this condition in humans.
Using functional MRI in a large multisite sample of more that 1,000 patients, four distinct neurophysiological biotypes of depression are defined. These biotypes are used to develop diagnostic classifiers that distinguish patients with depression from controls in separate multisite validation and replication cohorts, and can predict patient responsiveness to therapy.
The Wnt pathway inhibitor Dkk1, which is produced by bone marrow osteolineage cells, promotes hematopoietic recovery after radiation injury by both direct effects on hematopoietic cells and indirect effects on bone marrow endothelial cells.
Akkermansia muciniphila, a member of the gut microbiome, has been shown to improve metabolism in mice. Here it is reported that its pasteurization further improves this effect, and that one of its membrane proteins by itself has a similar benefit.
The thalamus-enriched microRNA miR-338-3p is depleted in mouse models of 22q11.2 deletion syndrome and in humans with schizophrenia, leading to a late-onset dysfunction of auditory thalamocortical synaptic transmission, behavioral abnormalities and altered sensitivity to antipsychotics.
By reducing the availability of extracellular L-cyst(e)ine, an engineered enzyme inhibits glutathione production and cripples antioxidant defenses of tumors in a variety of mouse models.
A genome-wide CRISPR screen reveals that FZD5, but none of the other nine Frizzled receptors encoded in the human genome, is a therapeutic vulnerability of pancreatic and colorectal tumors bearing RNF43 mutations.
Organoids formed by combining pluripotent-stem-cell-derived human neural crest cells with pluripotent-stem-cell-derived intestinal tissue show functional interstitial cells of Cajal and undergo waves of contraction; these tissues reveal insights into the molecular defects characterizing Hirschsprung's disease.
Copy-number alterations detected in circulating tumor cells at time of diagnosis predict chemosensitive versus chemorefractory responses; however, CTCs obtained after subsequent relapse bear a chemosensitive copy-number alteration profile, which suggests that different mechanisms drive initial and acquired chemoresistance.
Ram Savan and colleagues report that two miRNAs known to suppress type 3 interferon (IFN) signaling also downregulate type 1IFN signaling in hepatitis C virus (HCV)-infected hepatocytes. The findings provide insights into the mechanisms by which antiviral IFN signaling is inhibited in HCV infection.
Blockade of cIAP1 and cIAP2 induces a tumor cell-autonomous type-I IFN response that activates myeloid cells and potentiates anti-tumor immunity in pre-clinical models and patients with multiple myeloma.
AML cells carrying R882 mutations in DNMT3A fail to sense and repair DNA damage induced by standard-dose chemotherapy as a result of impaired chromatin remodeling
Using induced pluripotent stem cells from patients with familial dysautonomia, the authors show that in vitro models can recapitulate patient-specific differences in disease severity.
Spermidine, a naturally occurring polyamine, extends the lifespan of mice and is cardioprotective in both aged mice and hypertensive rats. In humans, high dietary spermidine intake is associated with reduced blood pressure and a lower incidence of cardiovascular disease.
A recent study confirms an association between vessel co-option and resistance to bevacizumab, an anti-vascular endothelial growth factor-A (VEGFA) antibody, in patients with liver metastases. The authors suggest a combined therapeutic strategy that reduces co-option in mice.