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Inhibition of CSF-1R with recurrent activating alterations and other actionable targets identified by genomic sequencing elicits clinical activity in patients with histiocytosis.
A unique case from the Colombian cohort of autosomal dominant Alzheimer’s disease is reported in which disease progression is substantially delayed despite unusually high amyloid plaque pathology, possibly related to a rare mutation in APOE3.
This first-in-human study assessed the feasibility of vaginal microbiome transplantation from healthy donors as a therapeutic alternative for patients suffering from symptomatic, intractable and recurrent bacterial vaginosis.
Individuals homozygous for the CCR5-∆32 allele have a 21% increase in mortality rate in the UK Biobank cohort. In light of the CRISPR-baby scandal, this work highlights the need for understanding the unintended consequences of introducing mutations in humans.
A deep residual learning framework identifies microsatellite instability in histology slides from patients with cancer and can be used to guide immunotherapy.
AAV-mediated CRISPR/Cas9 therapy extends life span and ameliorates disease-related phenotypes in a mouse model of Hutchinson–Gilford progeria syndrome.
An algorithm trained on half a million electronic medical records predicts chronic kidney disease in diabetic patients using a small set of defined clinical features, outperforming predictions derived from clinical trial data.