Featured
-
-
Article
| Open Access
Precision-guided treatment in high-risk pediatric cancers
In the observational ZERO Childhood Cancer Precision Medicine Program PRecISion Medicine for Children with Cancer (PRISM) trial, children with high-risk cancer were treated with molecular tumor board-recommended therapies, resulting in overall clinical response rates that translated into survival benefit after long-term follow-up.
- Loretta M. S. Lau
- , Dong-Anh Khuong-Quang
- & David S. Ziegler
-
Article
| Open Access
CD40 ligand antagonist dazodalibep in Sjögren’s disease: a randomized, double-blinded, placebo-controlled, phase 2 trial
In a phase 2 trial of dazodalibep, a CD40 ligand, with a crossover stage in two distinct populations of patients with Sjögren’s disease, the compound was well tolerated and led to significantly improved disease activity.
- E. William St. Clair
- , Alan N. Baer
- & Ilias Alevizos
-
Article
| Open Access
Bilateral gene therapy in children with autosomal recessive deafness 9: single-arm trial results
An interim analysis of a single-arm trial in 5 children with hereditary deafness shows that binaural AAV gene therapy is safe and leads to hearing improvement up to 13–26 weeks of follow-up.
- Hui Wang
- , Yuxin Chen
- & Yilai Shu
-
Research Briefing |
Heritable and spatial immunogenic traits co-predict the efficacy of immunotherapy in kidney cancer
A machine learning-derived gene signature that integrates germline HLA characteristics with a distinct spatial community of CD8+ T cells and tumor-associated macrophages showing immunogenic interactions is predictive of the response to immunotherapy in patients with advanced kidney cancer.
-
Article
| Open Access
Isatuximab, lenalidomide, dexamethasone and bortezomib in transplant-ineligible multiple myeloma: the randomized phase 3 BENEFIT trial
In patients with newly diagnosed, transplant-ineligible multiple myeloma, addition of weekly bortzomib to isatuximab, lenalidomide and dexamethasone leads to increased minimal residual disease negativity compared with isatuximab, lenalidomide and dexamethasone.
- Xavier Leleu
- , Cyrille Hulin
- & Thierry Facon
-
Article |
Claudin18.2-specific CAR T cells in gastrointestinal cancers: phase 1 trial final results
In the largest study so far of CAR T cells in patients with solid tumors, autologous CLDN18.2-targeting CAR T cell therapy was well tolerated in patients with advanced CLDN18.2-positive gastrointestinal cancers, with an overall response rate of 42.2% and a disease control rate of 91.1%.
- Changsong Qi
- , Chang Liu
- & Lin Shen
-
Article
| Open Access
Trastuzumab deruxtecan versus trastuzumab emtansine in HER2-positive metastatic breast cancer: long-term survival analysis of the DESTINY-Breast03 trial
Secondary and selected exploratory endpoints of the DESTINY-Breast03 trial of trastuzumab deruxtecan or trastuzumab emtansine in patients with advanced HER2-positive breast cancer are reported, including updated efficacy and safety data after a median follow-up of 41 months.
- Javier Cortés
- , Sara A. Hurvitz
- & Erika Hamilton
-
Article |
Pembrolizumab for advanced urothelial carcinoma: exploratory ctDNA biomarker analyses of the KEYNOTE-361 phase 3 trial
In an exploratory analysis of the Keynote-361 phase 3 trial that tested immunotherapy and chemotherapy in advanced urothelial carcinoma, different early ctDNA dynamics were observed between the two treatments, and ctDNA levels were associated with response to pembrolizumab, but not to chemotherapy. Anna Maria Ranzoni, in collaboration with the Nature Medicine team.
- Thomas Powles
- , Yen-Hwa Chang
- & Seyda Gunduz
-
Article
| Open Access
Inhibition of lysine acetyltransferase KAT6 in ER+HER2− metastatic breast cancer: a phase 1 trial
In this phase 1 trial, treatment of patients with ER+HER2− metastatic breast cancer with a selective catalytic inhibitor of the lysine acetyltransferase KAT6 shows a manageable safety profile and encouraging preliminary clinical efficacy.
- Toru Mukohara
- , Yeon Hee Park
- & Patricia M. LoRusso
-
Article |
Fruquintinib plus paclitaxel versus placebo plus paclitaxel for gastric or gastroesophageal junction adenocarcinoma: the randomized phase 3 FRUTIGA trial
In a randomized, double-blind, placebo-controlled phase 3 trial, the anti-vascular endothelial growth factor receptor small-molecule inhibitor fruquintinib plus chemotherapy significantly extended progression-free survival, but not overall survival, as second-line therapy in patients with advanced gastric or gastroesophageal junction cancers.
- Feng Wang
- , Lin Shen
- & Rui-Hua Xu
-
Brief Communication
| Open Access
Mutations in the U4 snRNA gene RNU4-2 cause one of the most prevalent monogenic neurodevelopmental disorders
Mutations in a small noncoding gene called RNU4-2 are responsible for one of the most common neurodevelopmental disorders that is mediated by a single gene.
- Daniel Greene
- , Chantal Thys
- & Ernest Turro
-
Article
| Open Access
Three-year outcomes of post-acute sequelae of COVID-19
Analyses from the US Department of Veterans Affairs databases reported residual elevated risk and health burden of long COVID at 3 years in hospitalized individuals after SARS-CoV-2 infection.
- Miao Cai
- , Yan Xie
- & Ziyad Al-Aly
-
Research Highlight |
A surprising abundance of pancreatic pre-cancers
AI-based three-dimensional genomic mapping reveals a large abundance of cancer precursors in normal pancreatic tissue — prompting new insights and research directions.
- Karen O’Leary
-
Research Highlight |
Affordable and simplified whole-body MRI
A whole-body scanner developed using a permanent 0.05-tesla magnet and deep learning has demonstrated its versatility in imaging various anatomical structures, showcasing its potential to address unmet clinical needs.
- Sonia Muliyil
-
Correspondence |
Generating clinical evidence for treatment and prevention options for long COVID
- Stephanie Buchholz
- , Eugenia Di Meco
- & Marco Cavaleri
-
Article |
Noninvasive assessment of organ-specific and shared pathways in multi-organ fibrosis using T1 mapping
T1 mapping noninvasively assesses fibrosis in multiple organs and enables risk stratification of mortality and provides insights into shared and organ-specific pathways underlying fibrosis.
- Victor Nauffal
- , Marcus D. R. Klarqvist
- & Patrick T. Ellinor
-
Research Highlight |
Half a century of immunization success
A modeling study marking 50 years of vaccination through the Expanded Programme on Immunization of the World Health Organization highlights its lifesaving impact on infant and child health.
- Sonia Muliyil
-
Research Briefing |
Immune signatures of disease stages and outcomes in myocardial infarction
A longitudinal multiomic dataset was assembled to characterize the immune landscape in myocardial infarction and chronic coronary syndromes. Multiomics factor analysis (MOFA) revealed immune signatures that associate with disease stage or treatment outcomes. This work opens new directions for future mechanistic and clinical studies on coronary artery disease and myocardial infarction.
-
Article
| Open Access
Prediction of recurrence risk in endometrial cancer with multimodal deep learning
A multimodal deep learning prognostic model based on histopathology outperforms current gold standards for identifying patients with endometrial cancer with different outcomes, in multiple external validation cohorts.
- Sarah Volinsky-Fremond
- , Nanda Horeweg
- & Tjalling Bosse
-
Article |
Cadonilimab with chemotherapy in HER2-negative gastric or gastroesophageal junction adenocarcinoma: the phase 1b/2 COMPASSION-04 trial
In the COMPASSION-04 trial, first-line treatment with a bispecific antibody targeting CTLA-4 and PD-1 and chemotherapy in patients with locally advanced or metastatic gastric or gastroesophageal junction adenocarcinoma resulted in clinical responses with a manageable safety profile.
- Xiangyu Gao
- , Ke Ji
- & Jiafu Ji
-
Article |
A spatial architecture-embedding HLA signature to predict clinical response to immunotherapy in renal cell carcinoma
Multiomics and spatial mapping of tumor samples derived from a real-world cohort of patients with advanced renal cell carcinoma, as well as integration of transcriptomics and human leukocyte antigen genotyping data, provides a machine learning-derived signature of response to immune checkpoint blockade.
- Lisa Kinget
- , Stefan Naulaerts
- & Abhishek D. Garg
-
Article
| Open Access
Multiomic analyses uncover immunological signatures in acute and chronic coronary syndromes
Multiomic factor analysis of blood multiomic data, including single-cell transcriptomics, for individuals with either acute or chronic coronary syndrome identifies immune cell signatures that correlate with treatment outcomes.
- Kami Pekayvaz
- , Corinna Losert
- & Konstantin Stark
-
Research Highlight |
In vivo gene editing for inherited vision loss
An early-phase trial suggests safety and improvements in vision after treatment with the CRISPR-based therapy EDIT-101, providing proof of concept for in vivo retinal gene editing.
- Karen O’Leary
-
Article
| Open Access
Non-invasive spinal cord electrical stimulation for arm and hand function in chronic tetraplegia: a safety and efficacy trial
Externally applied electrical stimulation over the cervical spinal cord improves arm and hand functions in people with chronic tetraplegia due to spinal cord injury.
- Chet Moritz
- , Edelle C. Field-Fote
- & Gregoire Courtine
-
Article |
Prediction of DNA methylation-based tumor types from histopathology in central nervous system tumors with deep learning
A deep learning model is used to classify central nervous system tumors based on their DNA methylation profile directly from histopathology, and showed high accuracy in a large set of external validation cohorts, potentially informing downstream treatment.
- Danh-Tai Hoang
- , Eldad D. Shulman
- & Kenneth Aldape
-
Article
| Open Access
p75 neurotrophin receptor modulation in mild to moderate Alzheimer disease: a randomized, placebo-controlled phase 2a trial
A phase 2a trial of LM11A-31 in mild to moderate Alzheimer disease suggests that p75NTR modulation is safe and attenuates measures of degeneration.
- Hayley R. C. Shanks
- , Kewei Chen
- & Taylor W. Schmitz
-
Article
| Open Access
A prognostic neural epigenetic signature in high-grade glioma
A neural epigenetic signature detectable via plasma analyses is prognostic in patients with glioblastoma, resembling an oligodendrocyte-progenitor- and neuronal-progenitor-cell-like state and showing increased neuro-to-glioma synapse formation.
- Richard Drexler
- , Robin Khatri
- & Franz L. Ricklefs
-
Article |
Integrative multi-omics profiling in human decedents receiving pig heart xenografts
Multi-omics profiling of the blood and heart of two human decedents receiving pig heart xenografts, including single-cell studies, reveals early immune responses and perioperative cardiac xenograft dysfunction in one of the two decedents, which may be due to mismatched heart size and/or insufficient immunosuppression.
- Eloi Schmauch
- , Brian Piening
- & Brendan J. Keating
-
Article |
Prevalence of comorbidities in individuals with neurodevelopmental disorders from the aggregated phenomics data of 51,227 pediatric individuals
Data from pediatric populations with neurodevelopmental disorders, obtained through a combinatorial strategy of literature review scoping and in-patient appointments, were used to construct a Phenomics Aggregation Database (PhenomAD-NDD) that can aid clinical diagnosis of comorbidities.
- Alexander J. M. Dingemans
- , Sandra Jansen
- & Bert B. A. de Vries
-
Article
| Open Access
Trastuzumab deruxtecan in HER2-positive advanced gastric cancer: exploratory biomarker analysis of the randomized, phase 2 DESTINY-Gastric01 trial
Exploratory analyses of the DESTINY-Gastric01 trial show that baseline HER2-associated biomarkers in circulating tumor DNA or tissue samples were associated with therapeutic response in patients with HER2-positive tumors, and these analyses identify potential drivers of resistance.
- Kohei Shitara
- , Yung-Jue Bang
- & Kensei Yamaguchi
-
Article
| Open Access
Progranulin AAV gene therapy for frontotemporal dementia: translational studies and phase 1/2 trial interim results
Interim results from the low-dose cohort of the PROCLAIM trial show that AAV-based delivery of the progranulin gene in patients with frontotemporal dementia was safe and was associated with increased CSF progranulin.
- Jeffrey Sevigny
- , Olga Uspenskaya
- & Franz Hefti
-
Research Highlight |
Gut bacteria switch A and B blood types to ‘universal’
Enzymes produced by Akkermansia muciniphila generated group O blood by degrading A and B antigens, which could help solve blood donor shortages in the future.
- Karen O’Leary
-
Article
| Open Access
Association between pretreatment emotional distress and immune checkpoint inhibitor response in non-small-cell lung cancer
In a prospective observational study, symptoms of anxiety and/or depression were associated with worse response to first-line treatment with immunotherapy in patients with advanced non-small-cell lung cancer.
- Yue Zeng
- , Chun-Hong Hu
- & Fang Wu
-
Article
| Open Access
Screening and diagnosis of cardiovascular disease using artificial intelligence-enabled cardiac magnetic resonance imaging
A two-step, video-based deep learning model is developed to first screen for cardiac anomalies using noncontrast magnetic resonance imaging, followed by diagnosis of 11 types of cardiovascular disease using gadolinium enhancement-based imaging.
- Yan-Ran (Joyce) Wang
- , Kai Yang
- & Shihua Zhao
-
Review Article |
Artificial intelligence in surgery
This Review outlines the state of the art of artificial intelligence in the preoperative, intraoperative and postoperative settings, where it has enormous potential to improve patient outcomes, surgical education and system efficiencies.
- Chris Varghese
- , Ewen M. Harrison
- & Eric J. Topol
-
Article
| Open Access
High-resolution African HLA resource uncovers HLA-DRB1 expression effects underlying vaccine response
Genome-wide analyses of vaccine antibody responses in 2,499 infants from Uganda, South Africa and Burkina Faso identify associations between specific HLA genes and response to eight vaccines, providing insights that could be considered for population-adjusted vaccine design strategies.
- Alexander J. Mentzer
- , Alexander T. Dilthey
- & Manjinder S. Sandhu
-
Research Briefing |
Studying paired patient tissue and blood enables insights into immunotherapy toxicity
Using single-cell RNA and T cell receptor sequencing along with microscopy, we identified the cell types and genes associated with immune checkpoint inhibitor therapy-related colitis. Our study will help to identify targets for early diagnosis and lays the groundwork for the development of safer immunotherapy regimens.
-
Research Highlight |
Gene therapy trials deliver double win
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.
- Karen O’Leary
-
Article |
Single-cell transcriptomic analyses reveal distinct immune cell contributions to epithelial barrier dysfunction in checkpoint inhibitor colitis
Single-cell multi-omic analysis of 300,000 cells from 29 patients representing peripheral immune cells and colon mucosal immune, epithelial and mesenchymal cells reveals crosstalk between circulating and tissue-resident immune cells with epithelial cells in checkpoint inhibitor colitis and identifies potential therapeutic targets.
- Molly Fisher Thomas
- , Kamil Slowikowski
- & Alexandra-Chloé Villani
-
Comment |
An updated framework for SARS-CoV-2 variants reflects the unpredictability of viral evolution
The World Health Organization framework for tracking SARS-CoV-2 variants has been updated to reflect the continued evolution of the virus; this framework could be adapted for other emerging respiratory diseases with epidemic and pandemic potential.
- Lorenzo Subissi
- , James Richard Otieno
- & Maria D. Van Kerkhove
-
Article
| Open Access
Effects of a personalized nutrition program on cardiometabolic health: a randomized controlled trial
A randomized controlled trial showed that following a personalized dietary program led to significant improvements in cardiometabolic and gut health as well as reductions in body weight compared to following standard dietary advice according to US Department of Agriculture guidelines.
- Kate M. Bermingham
- , Inbar Linenberg
- & Sarah E. Berry
-
Research Briefing |
Exome sequencing in a Chinese cohort of children with cerebral palsy identifies likely pathogenic variants
Exome-sequencing analysis in a Chinese cohort of 1,578 children with cerebral palsy established a genetic etiology in 387 of the patients (24.5%). Children with cerebral palsy for whom perinatal asphyxia had been recorded at birth were found to be more likely to carry pathogenic or potentially pathogenic variants.
-
News & Views |
APOE4 homozygosity is a new genetic form of Alzheimer’s disease
New data confirm that APOE4 homozygosity is a major genetic cause of Alzheimer’s disease, warranting the development of specialized research strategies, treatment approaches and clinical trials.
- Qin Xu
- , Zherui Liang
- & Yadong Huang
-
Article
| Open Access
Pemigatinib in previously treated solid tumors with activating FGFR1–FGFR3 alterations: phase 2 FIGHT-207 basket trial
In a tumor-agnostic phase 2 basket trial, the oral FGFR1–FGFR3 inhibitor pemigatinib elicits responses in tumor types beyond cholangiocarcinoma and bladder cancer and in tumor types with rarer FGFR alterations, with insights provided into resistance mechanisms.
- Jordi Rodón
- , Silvia Damian
- & Lipika Goyal
-
News & Views |
Adapting vision–language AI models to cardiology tasks
Vision–language models can be trained to read cardiac ultrasound images with implications for improving clinical workflows, but additional development and validation will be required before such models can replace humans.
- Rima Arnaout
-
Article |
Exome sequencing reveals genetic heterogeneity and clinically actionable findings in children with cerebral palsy
Using exome sequencing data from one of the largest cohorts of children with cerebral palsy, the genetic diagnostic rates of single-nucleotide and copy number variants were assessed and a sizeable fraction found to be clinically actionable.
- Yangong Wang
- , Yiran Xu
- & Qinghe Xing
-
Research Highlight |
Sustainable, low-cost cancer diagnostics
Researchers have developed a low-resource, environmentally friendly cancer-diagnosis platform that detects metabolites in dried serum spots.
- Karen O’Leary
-
News & Views |
The search for authentic microbiome–disease relationships
A strategy that controls confounders in quantitative microbiome data challenges the validity of previously reported microbial markers in colorectal cancer and serves as a wake-up call for the microbiome research field.
- Na Jiao
- , Lixin Zhu
- & Ruixin Zhu
-
Article |
Correlates of protection against symptomatic SARS-CoV-2 in vaccinated children
The dynamics and durability of immune responses associated with protection against symptomatic infection in children offer insights to guide vaccination policies in pediatric populations.
- Youjia Zhong
- , Alicia Y. H. Kang
- & Eng Eong Ooi
Browse narrower subjects
- Biochemistry
- Biological techniques
- Biophysics
- Biotechnology
- Cancer
- Cell biology
- Chemical biology
- Computational biology and bioinformatics
- Developmental biology
- Drug discovery
- Ecology
- Evolution
- Genetics
- Immunology
- Microbiology
- Molecular biology
- Neuroscience
- Physiology
- Plant sciences
- Psychology
- Stem cells
- Structural biology
- Systems biology
- Zoology
