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iPSC-derived motor neurons from over 30 heterogeneous sporadic ALS cases exhibit pathologies correlated with clinical disease progression, are more similar to FUS/TDP-43 familial ALS than SOD1-ALS and are corrected by repurposing of ropinirole.
An algorithm-selected gene signature focused on tumor immune evasion and suppression predicts response to immune checkpoint blockade in melanoma, exceeding the accuracy of current clinical biomarkers.
CRISPR–Cas9-mediated gene editing of TSC1 and TSC2 in human pluripotent stem cells is used to investigate the contribution of tuberous sclerosis complex–mechanistic target of rapamycin complex 1 signaling to human neural development in two-dimensional monolayer and three-dimensional spheroid models of the neurodevelopmental disorder tuberous sclerosis complex.
Patients with glioblastoma experience lymphopenia and sequestration of T cells in the bone marrow, which is recapitulated in mice with brain tumors, where the reversible nature of this effect is demonstrated by an approach that enables the efficacy of other immunotherapeutics.
A novel deep learning architecture performs device-independent tissue segmentation of clinical 3D retinal images followed by separate diagnostic classification that meets or exceeds human expert clinical diagnoses of retinal disease.
A blood-based DNA sequencing assay to infer tumor mutational burden in the absence of tumor biopsy predicts response to PD-L1 blockade in patients with non-small-cell lung cancer.
A bacterial screen yields a Cas9 variant that retains high on-target activity when delivered in the RNP format. As proof of principle, this Cas9 variant enables high-level correction of the sickle cell disease mutation in patient-derived HSPCs.
Mechanism-based small-molecule inhibitors targeting a gut microbial enzyme lower circulating levels of the prothrombotic metabolite trimethylamine-N-oxide and suppress diet-induced thrombosis in mice.
Early and prolonged administration of antiretroviral therapy to SIV-infected and post-exposure-vaccinated rhesus macaques was associated with absence or delay of detectable virus after therapy interruption.
The muscle-secreted, exercise-induced peptide hormone apelin decreases with aging and sarcopenia, and its repletion in aged mice with recombinant protein improves muscle mass and function.
Oncogenic NOTCH1 controls transcriptional activation of the heat shock response in T cell acute lymphoblastic leukemia and uncovers potential biomarkers of sensitivity to HSP90 inhibition.
Interleukin-1β promotes an atheroprotective phenotype in late-stage lesions of mice, suggesting the possibility of deleterious effects of interleukin-1β blockade in the setting of myocardial infarction.
Topographic analysis of the active regulatory landscape in estrogen receptor-positive breast cancer uncovers a role for transcription factor YY1 in modulating phenotypic heterogeneity during tumor evolution and endocrine resistance.
Using 13C-labeled substrates in vivo, this group shows that metformin inhibits mG3PDH to reduce hepatic gluconeogenesis and lower glycemia by altering the redox potential of the cytosol of hepatocytes rather than affecting substrate availability.
Microsatellite instability and Epstein–Barr virus positivity represent novel biomarkers of clinical response to PD-1 blockade in patients with metastatic gastric cancer.
Mapping of the clonal structure of bone marrow cells in patients with acute myeloid leukemia treated with the IDH2 inhibitor enasidenib reveals heterogeneity in the cellular differentiation response and in mechanisms of relapse.
Transfer of senescent cells into naive, young mice can induce physical dysfunction, and a senolytic can reverse this dysfunction and potently increase lifespan in aged mice.