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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.
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.
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.
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.
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 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.