Neuroimaging and cerebrospinal fluid analyses in humans reveal that loss of blood–brain barrier integrity and brain capillary pericyte damage are early biomarkers of cognitive impairment that occur independently of changes in amyloid-β and tau.

A deep-learning algorithm, trained on over 17,000 real-world patient facial images, achieves high accuracy in identifying rare genetic disorders.

A deep learning algorithm applied to the electrocardiogram—a test of the heart’s electrical activity—can detect abnormally low contractile function of the heart, opening up the possibility for a simple screening tool for this condition.

Analysis of electrocardiograms using an end-to-end deep learning approach can detect and classify cardiac arrhythmia with high accuracy, similar to that of cardiologists.

A novel chimeric antigen receptor (CAR) T cell design improves therapeutic efficacy by balancing effector and memory profiles.

The new RAF inhibitor PLX8394 selectively blocks ERK signaling in tumors driven by class 1 and/or class 2 BRAF mutations and BRAF fusions while maintaining a broad therapeutic window.

RNA editing enzyme ADAR1 is a therapeutic vulnerability in tumors with constitutive cell-autonomous production of interferon-stimulated genes

High-resolution sequencing of longitudinal patient samples reveals subclonal mutational diversity in the stem cell compartment driving parallel evolution patterns in acute myeloid leukemia progression.

Intratumoral T cells that are capable of recognizing adjacent tumor tissue antigens can be exceedingly rare.

Nongenetic activation of Aurora kinase A in the majority of patients with non-small-cell lung cancer mediates adaptive resistance to EGFR inhibition and offers an opportunity for combination treatment.

Initial results from a first-in-human study show that PET imaging with PD-L1 antibodies outperforms immunohistochemistry- or RNA-sequencing-based biomarkers for prediction of clinical response to immunotherapy.

A new population of T helper cells employs unique signals to support autoreactive B cells in patients with systemic lupus erythematosus.

Single-cell RNA-seq of a mouse model of multiple sclerosis uncovers new oligodendrocyte populations and putative disease markers and suggests new mechanisms underlying the pathogenesis of the disease.

Skin from individuals with a rare immunodeficiency harbors more eukaryotic viruses than healthy skin, highlighting the role of immune surveillance in modulating the skin microbiome.

Radiotherapy-induced abscopal responses enhance the efficacy of anti-CTLA-4 in patients with non-small-cell lung cancer.

Infant nutrition and maternal weight gain during pregnancy impact early-life acquisition and function of the gut microbiome .

T cells that react to SpCas9 are observed in the peripheral blood mononuclear cells isolated from healthy humans. These findings may have implications for CRISPR–Cas9 therapeutics.

A new bioinformatic tool identifies a candidate source of bloodstream infection for better management and prevention of hospital-acquired infections.

AAV-mediated base editing corrects an autosomal recessive mutation in the Pah^enu2 gene and ameliorates molecular deficits in a mouse model of metabolic liver disease.

A biocompatible device built from naturally dissolving components and controlled by wireless technology enables programmable electrical stimulation of injured rodent peripheral nerves to accelerate regeneration and recovery.

Viral-mediated base editing in utero enables therapeutic editing of two metabolic genes in mice.

Neoadjuvant combination treatment with nivolumab and ipilimumab in patients with high-risk melanoma results in higher response rates than nivolumab monotherapy and warrants future optimization of dosing regimens to preserve efficacy while limiting toxicity.

The long-term follow-up results of a phase 1/2 retinal gene therapy clinical trial for choroideremia ( NCT01461213 ) support the safety and efficacy of the treatment.

Large tumors induce anemia and expansion of CD45⁺ immature erythroid cells, which represent a major immunosuppressive population in the spleen, contributing to systemic suppression of T cell immunity in late-stage cancer.

Neoadjuvant combination immunotherapy in patients with advanced melanoma shows favorable activity over adjuvant treatment and warrants future evaluation with modified dosing schedules to reduce treatment-related adverse events.

In vivo tracking of hematopoietic stem and progenitor cells in humans treated with lentiviral gene therapy unveils the resilience of hematopoietic stem cells and the long-term survival of lymphoid-biased precursors.

Combination of two broadly neutralizing antibodies is effective in reducing HIV-1 viremia and in limiting the emergence of resistant viral variants in individuals harboring antibody-sensitive viruses.

Mitochondrially targeted zinc-finger nucleases reduce mutational burden and correct biochemical defects in a mouse model of mitochondrial disease.

Intracortical activity data recorded over 2 years in a tetraplegic patient is used to develop an artificial intelligence algorithm that achieves fast, accurate, and stable movement decoding to reenable real-time control of the paralyzed forearm.

In a human subject with chronic paraplegia, a combination of epidural electrical stimulation and long-term rehabilitative training have culminated in the first report of unassisted, voluntary independent stepping in a paralyzed individual.

Mitochondrial-targeted TALENs reduce mutation burden and correct biochemical defects in a mouse model of mitochondrial disease.

In pediatric brain tumors that are accompanied by epileptic seizures, the BRAF somatic mutation V600E contributes to intrinsic epileptic properties in neurons, which can be suppressed by vemurafenib in mice.

The definition of a 'healthy' microbiome is impacted by geographic regional variations.

Stool microbiota composition correlates with the ethnic backgrounds of people living in the same city, suggesting that geographical location and ethnicity have distinct effects on microbiota.

A gene signature identified in spontaneously regressing neuroblastoma identifies responders to immune checkpoint blockade among patients with melanoma with accuracy superior to previously reported biomarkers.

A deep-learning algorithm is developed to provide rapid and accurate diagnosis of clinical 3D head CT-scan images to triage and prioritize urgent neurological events, thus potentially accelerating time to diagnosis and care in clinical settings.

T cells kill blood-stage Plasmodium vivax, providing a rationale for the development of a T cell vaccine against this parasite.

Retroelements located in antisense orientation within interferon-regulated genes are reactivated in a subset of cancer cells and initiate a STING- and MAVS-dependent feed-forward inflammatory loop, driving antitumor immunity and exhaustion.

In utero GBA gene therapy extends lifespan and provides long-lasting phenotypic amelioration in a mouse model of neuronopathic Gaucher disease. Fetal ultrasound-guided in utero gene vector delivery is also achieved in the non-human primate brain.

A phase 2 placebo-controlled randomized trial reveals that verapamil promotes beta cell function in adult subjects with recent-onset type 1 diabetes.

A glutamate-gated chloride channel delivered via gene therapy is shown to detect elevated brain glutamate levels and trigger the suppression of neuronal excitability, thereby attenuating seizure activity in two rodent models of epilepsy.

Targeting a post-translational modification of Fas by recombinant Glrx reverses established lung fibrosis in a mouse model of age-related idiopathic pulmonary fibrosis.

Small-molecule inhibitors of the serine dipeptidases DPP8 and DPP9 block AML progression by promoting CARD8-dependent pyroptosis of leukemic myeloid cells.

Metformin reverses established lung fibrosis in a bleomycin model in mice.

Extensive, high-dimensional characterization of T cells in breast cancer reveals activated T_RM population and a gene signature associated with improved prognosis.

Indoleamine 2,3-dioxygenase normally suppresses inflammation, but knockout of its gene is metabolically beneficial as its depletion reshapes the gut microbiota.

Deep single-cell RNA sequencing of tumor-infiltrating T cells in non-small-cell lung cancer identifies features associated with functional states and clinical outcome

ALS/FTD-related C9orf72 dipeptide-repeat proteins inhibit protein translation and impair stress granule dynamics, and they cause motor and cognitive deficits in mice.

Agonism of microglial glucagon-like peptide-1 receptor (GLP1R) using a brain-penetrant peptide prevents the generation of neurotoxic astrocytes and ameliorates disease progression in two rodent models of Parkinson’s disease.

In vivo shRNA screens in sensitized genetic backgrounds identify p53-activated target genes involved in DNA repair that enable its tumor suppressor function.