Thank you for visiting nature.com. You are using a browser version with limited support for CSS. To obtain
the best experience, we recommend you use a more up to date browser (or turn off compatibility mode in
Internet Explorer). In the meantime, to ensure continued support, we are displaying the site without styles
and JavaScript.
Final results of two studies — whose preliminary data led to regulatory approval of the gene therapy exagamglogene autotemcel — describe highly effective treatment of sickle-cell disease and transfusion-dependent β-thalassemia.
A randomized controlled trial involving a telemedicine-based approach for the management of patients with acute coronary syndrome had several clinical benefits relative to standard of care.
An mRNA-based drug aims to replace a faulty enzyme and restore metabolic function in children with propionic acidemia — with encouraging early clinical results.
In a multicenter clinical trial, patients with early-stage Parkinson’s disease treated with lixisenatide, a drug currently used for the treatment of diabetes, showed improvement in their motor scores compared with those on placebo.
Researchers developed an AI model that designs novel, synthesizable antibiotic compounds — several of which showed potent in vitro activity against priority pathogens.
Global nephrology societies have called on the WHO and health communities to tackle the growing burden of chronic kidney disease, which has been under-recognized for too long.
Deployment of mobile vaccination teams to remote communities in Sierra Leone substantially increased COVID-19 vaccine uptake, and could potentially be bundled with other health interventions.
Patients with carotid artery plaque that contain microplastics and nanoplastics were found to be at higher risk of cardiovascular events and mortality than those in whom the particles were not detected.
In rhesus macaques, treatment with an IL-15 superagonist and broad neutralizing antibodies led to durable suppression of viremia after discontinuation of antiretroviral therapy.
People with type 2 diabetes who underwent bariatric surgery compared to medical or lifestyle interventions had better long-term outcomes, such as greater diabetes remission and less reliance on medication.
Longitudinal data from the China Cognition and Aging Study map the sequential biomarker changes that begin almost two decades before clinical onset of Alzheimer’s disease.
MEGA is a new CRISPR-based RNA-editing platform with the ability to enhance the fitness of CAR T cells; it may also overcome certain limitations of conventional DNA-targeting CRISPR–Cas9 systems.
In a head-to-head study, a multitarget version of the widely used FIT (fecal immunochemical test) showed a higher cancer detection rate than the standard test.
The Born in Guangzhou Cohort Study prospectively collects genetic and phenotypic data from thousands of families, providing insights into genetics, early-life exposures and long-term health.
An intervention that incorporates electronic pill boxes and remote adherence monitoring improved treatment success in patients with tuberculosis in Tibet — making this a promising strategy for low-resource settings.
Patients who received an in vivo CRISPR-based gene-editing therapy for hereditary angioedema showed no serious side effects and nearly complete disease control.
Neuroimaging data uncover dynamic trajectories associated with brain cortical changes in women during pregnancy and in the postpartum period that are influenced by the type of childbirth.
In patients with resected, high-risk melanoma, the addition of a personalized mRNA-based neoantigen therapy to adjuvant pembrolizumab was more effective than pembrolizumab alone.