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Amplification of wild-type KRAS in a subset of gastroesophageal tumors drives intrinsic resistance to MAPK inhibition that can be overcome by combined targeting of MEK and SHP2.
In a mouse model of intellectual disability, the chromatin remodeling protein ATR-X is shown to bind to G-quadruplex DNA structures and modulate target gene expression, which can be pharmacologically targeted to restore neuronal and cognitive phenotypes in these animals.
Inhibition of immune cell–derived acetylcholine synthesis or of its signaling via CHRNA2 in beige adipocytes reduces thermogenesis and exacerbates diet-induced obesity, suggesting a new mode of immuno–fat communication in energy metabolism.
An integrated resource of (epi)genomic features in annotated chronic lymphocytic leukemia (CLL) primary samples uncovers subgroup-specific regulatory alterations associated with clinical behavior.
Interactions between the MLL3 histone methyltransferase and the BAP1–UTX complex set the level of histone H3K27 methylation and suggest a new therapy for MLL3-mutant cancer.
Excess anti-Müllerian hormone during pregnancy results in polycystic ovary syndrome-like phenotypes in female offspring, possibly explaining its pathogenesis as well as suggesting a possible therapy.
For more than two decades, Nature Medicine has been the prime venue for publication of outstanding work in the translational space. Now the journal’s scope is evolving to embrace the clinical research that meets the challenges and complexities of contemporary medicine.
In triple-negative breast cancer, therapeutic response to carboplatin and docetaxel is similar. However, carboplatin therapy is superior to docetaxel in patients with germline BRCA1 or BRCA2 mutations, but ‘BRCAness’ does not predict sensitivity to carboplatin.
An artificial intelligence (AI) using a deep-learning approach can classify retinal images from optical coherence tomography for early diagnosis of retinal diseases and has the potential to be used in other image-based medical diagnoses.
Anti-GD2 chimeric antigen receptor (CAR)-modified T cells may be a new and innovative approach for the treatment of pediatric H3-K27M-mutant diffuse midline gliomas.
A crucial role for glucose metabolism has been identified in wound repair and in the pathogenesis of chronic inflammatory skin diseases, indicating that targeting metabolism is an approach for treating psoriasis.
Expression of PM20D1 is regulated by long-range chromatin interactions with an Alzheimer’s disease risk haplotype, and PM20D1 overexpression reduces AD-like pathology and cognitive impairment in a rodent model.
The transcription factor ELK-1 is upregulated in patients with major depressive disorder, and selective inhibition of hippocampal ELK-1 produces rapid antidepressive effects in rodent models of depression.