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Initial results from phase I/II lentiviral gene therapy trials provide early evidence supporting its safety and efficacy in treating patients with X-linked chronic granulomatous disease.
CRISPR–Cas9-mediated gene editing restores dystrophin expression in both pig and human induced pluripotent stem cell models of Duchenne muscular dystrophy, with beneficial effects on skeletal muscle and cardiac function.
Inactivating mutations in a protein kinase, NEK10, cause a genetic bronchiectasis syndrome in humans that is characterized by short motile cilia and impaired mucociliary transport.
Most infants with cystic fibrosis have poor early linear growth in their first year despite nutritional supplementation and treatment. Intestinal dysbiosis in these infants is associated with low length, suggesting a path for intervention.
Results from an expansion cohort of the PROFILE 1001 trial describe the anti-tumor activity of crizotinib in people with non-small-cell lung cancer harboring a MET exon 14 alteration.
A retrospective analysis of existing computed tomography scans shows the feasibility of an automated process for evaluating osteoporotic fracture risk that could be used as an initial screening tool when FRAX inputs are unavailable.
Leveraging the availability of nationwide electronic health records from over 500,000 pregnancies in Israel, a machine-learning approach offers an alternative means of predicting gestational diabetes at high accuracy in the early stages of pregnancy.
Bayesian spatio-temporal modeling of mortality from injuries in the contiguous USA shows increases in the number of deaths attributable to abnormal temperature fluctuations due to global heating.
Expansion of pathogenic Candida species coupled with bacterial dysbiosis in the gut precedes Candida bloodstream infections in hematopoietic cell transplant recipients.
A prospective, multicenter, case–control clinical trial evaluates the potential of artificial intelligence for providing accurate bedside diagnosis of patients with brain tumors.
Analysis of a mass cytometry dataset for different human solid tumors coupled with murine reverse translational experiments suggests that targeting CD73 could enhance the efficacy of checkpoint inhibitor therapy in glioblastoma.
Aptamer-based proteomic analysis of plasma from healthy individuals aged 18–95 years reveals wave-like patterns of protein expression that are associated with age-related diseases and phenotypic traits.
Analysis of known viruses in stool samples from young children with high genetic risk for type 1 diabetes shows that sustained enterovirus B (EV-B) infections, rather than independent, short-duration EV-B infections, might be involved in the development of islet autoimmunity, but not type 1 diabetes.
Large-scale aptamer-based scanning of plasma proteins coupled with machine learning demonstrates proof-of-concept and feasibility of an individualized health check using a single blood sample.
The degree of sequence divergence between patient MHC class I alleles influences the response to immune checkpoint blockade therapy independently of tumor mutational burden.
Large-scale sequencing of coding exons of MRAP2 in 9,418 adults and adolescents identifies loss-of-function mutations that are associated with monogenic obesity, hypertension and hyperglycemia.