This was a prospective, single-arm, open-label pilot trial. The primary end point was 6-month progression-free survival (PFS). Disease progression was assessed according to the Response Assessment in Neuro-Oncology criteria by independent radiological review. Radiological response was evaluated using fluid-attenuated inversion-recovery sequences to compare FUS-exposed vs nonexposed regions. Plasma cell–free DNA (cfDNA) concentrations were measured before and after FUS treatment.

RESULTS:

Between July 2020 and August 2023, 6 patients received a median of 14.5 sessions of biweekly FUS-BEV (10 mg/kg). The median PFS was 11 months, with a 6-month PFS rate of 66.7%. The only FUS-related adverse event was transient scalp heating (grade 1; 1.9%). A fluid-attenuated inversion recovery normalization effect emerged within 1 month after treatment. Plasma cfDNA increased significantly post-FUS, with total cfDNA rising 2.03 ± 0.76-fold, EGFR cfDNA 1.77 ± 0.76-fold, and HMBS cfDNA 1.68 ± 0.66-fold.

New research has shown that a genome editing technique can be used to alter a single gene in human embryonic cells, enabling the study of very early human development in unparalleled detail.

The technique, called base editing, is a more precise version of the genome editing technique CRISPR/Cas9. It can change a single nucleotide base pair — the basic building block of DNA — within a human genome of approximately 3 billion base pairs.

Using base editing, the researchers blocked a gene called NANOG in very early-stage human embryos, and found that the cells of the early embryo could not develop into more specialised pluripotent cells called the epiblast — which later form the body.

This study presents an human induced pluripotent stem-cell-based culture model for studying cell state transitions during epithelial-to-mesenchymal transition.

BACKGROUND: SIRT1 (sirtuin 1), a nicotinamide adenine dinucleotide–dependent protein deacetylase.

Regulates cardiovascular inflammation by modulating cellular stress, inhibiting NLRP3.

(NLR family, pyrin domain-containing 3) activation, and promoting the clearance of.

Damaged mitochondria. However, its precise role in the pathogenesis of Kawasaki disease.

Leptomeningeal metastasis (LM) carries a poor prognosis in EGFR-mutant non-small cell lung cancer (NSCLC). This study aims to explore clinical features and survival outcomes and develop a nomogram for overall survival (OS) in this population.

We retrospectively enrolled 288 EGFR-mutant NSCLC patients with LM from Fujian Cancer Hospital (January 2019–March 2024). Prognostic variables were selected by LASSO regression followed by multivariable Cox proportional hazards modeling. Internal validation used 1000-iteration bootstrap resampling. Model performance was assessed by the time-dependent area under the receiver operating characteristic curve (AUC), calibration plots, and decision curve analysis.

Median OS was 16.2 months. Before LM, 71.5% of patients received third-generation EGFR tyrosine kinase inhibitors (TKIs), and 76.7% received third-generation EGFR-TKI therapy after LM. Eight variables were identified by LASSO-Cox regression: ECOG performance status (PS), prior lines of systemic therapy, cranial MRI findings, third-generation EGFR-TKI therapy before and after LM, antiangiogenic therapy after LM, chemotherapy after LM, and CNS radiotherapy after LM. Post-LM third-generation EGFR-TKI therapy was the strongest treatment-associated prognostic factor (HR = 2.23; p 0.001), with survival benefit maintained regardless of prior third-generation EGFR-TKI exposure (prior-exposed: HR = 0.60, p = 0.029; TKI-naïve: HR = 0.18, p 0.001). The nomogram demonstrated good discrimination (AUC: 0.777, 0.778, and 0.742 for 6-, 12-, and 18-month OS; C-index: 0.716).