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Engineering of a menthol-controlled gene switch whose components are all of human origin may facilitate clinical translation of this approach, in which therapeutic protein production is triggered by topical application of menthol to the skin.
Screening of Cas9 and gRNA combinations identifies the variant SaCas9-KKH that selectively disrupts the mutant Tmc1/TMC1 allele that causes hearing loss, prevents deafness and preserves inner-ear hair cell function in Tmc1 mutant Beethoven mice.
Local release of an anti-CD47 nanobody from an engineered non-pathogenic Escherichia coli strain is safe and enhances activation of tumor-infiltrating T cells, slowing tumor growth in mice.
Inhibition of histone and DNA methyltransferase activity enhances sensitivity to platinum-based and immunotherapy in a novel transgenic mouse model of metastatic bladder cancer.
Supplementation with Akkermansia muciniphila, a gut microbe previously associated with metabolic health in preclinical models, is safe and well tolerated in humans and may improve metabolic parameters in overweight and obese patients.
A recurrent gain-of-function mutation in ARAF causes lymphatic anomaly in two unrelated patients, and treating one of the patients with an FDA-approved MEK inhibitor resulted in a remarkable recovery, exemplifying the power of precision medicine.
Epigenetic states reminiscent of the cell of origin define clinically relevant markers for stratification of patients with pancreatic neuroendocrine cancer.
A closer look at the gut microbiome of elite marathon runners unveils a microbe-encoded enzymatic process that contributes to enhanced athletic performance.
Donor-derived, EBV-specific CD8+ T cells engineered to express a high-affinity WT1-specific TCR established persistent T cell responses that safely prevented post-HCT relapse in patients with high-risk AML.
Triple therapy combining BRAF and MEK inhibitors with immune checkpoint blockade may benefit a subset of patients with BRAFV600-mutated metastatic melanoma.
A randomized phase 2 trial testing triple combination of BRAF, MEK and PD-1 inhibition as first-line therapy in patients with BRAF-mutant melanoma shows durable responses and encouraging progression-free survival.
Treatment with BRAF and/or MEK inhibitors followed by addition of anti-PD-L1 in BRAF-mutant melanoma patients is safe and shows promising anti-tumor activity.
A diagnostic tool based on blood RNA-seq is shown to identify causal genes and variants linked to clinical phenotypes in individuals with rare diseases for which whole-exome genetic sequencing was uninformative.
Study of influenza virus transmission in humans provides evidence that hemagglutinin stalk-specific antibodies correlate with protection from infection.
A convolutional neural network performs automated prediction of malignancy risk of pulmonary nodules in chest CT scan volumes and improves accuracy of lung cancer screening.
A pick-the-winner clinical trial design in patients with metastatic triple-negative breast cancer shows that immune induction with doxorubicin or cisplatin may improve clinical responses to PD-1 blockade and induce a more favorable tumor microenvironment.