Journal of Clinical Investigation

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Ibrutinib is linked to hypertension in 225 chronic lymphocytic leukemia (CLL) patients, and in mice it increases arterial constriction via C/EBP-β–driven CaV1.2 upregulation.

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In hepatocytes, DOT1L acts as a key sensitizer to ferroptosis and subsequent liver injury, and DOT1L inhibition reverses this effect.

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Daily subcutaneous exaluren (0.75 mg/kg) for 8 weeks in three adolescents with autosomal recessive Alport syndrome with COL4A4 nonsense mutations increased collagen α3(IV), α4(IV), and α5(IV) abundance.

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ELF4 is a conserved, immune-enriched transcription factor in human kidney disease, linked to severity and expressed highest in NK cells.

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ATRX protein loss is linked to significantly worse outcomes in uterine leiomyosarcoma (53%), undifferentiated pleomorphic sarcoma (37%), and dedifferentiated liposarcoma (8%).

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A CD19/HIV dual CAR T strategy in a humanized mouse leukemia model eradicated tumors comparable to CD19-only CAR T, while a CD19.BBζ-IL-18 plus CD4.28ζ-C34-CXCR4* design enhanced HIV and tumor control.

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Decidua-enriched effector-like memory CD8+ T cells marked by CD45RA in humans and CX3CR1 in mice accumulate in the maternal-fetal interface via CX3CL1.

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Cellular senescence programs in facioscapulohumeral muscular dystrophy muscle is linked to DUX4 activity and clinical severity, including in STIR-negative prodystrophic tissue.

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Drug-controllable AAV-mediated KCNA1 expression normalized elevated neuronal firing and reduced spontaneous seizure burden in seizure-prone Scn2a-deficient mice on the DBA/2J background.

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Obesity (BMI ≥ 30) predicts improved survival after immune checkpoint inhibitor therapy only in male patients, with parallel benefit seen in diet-induced obesity male tumor-bearing mice.

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An early-stage synthetic immunological niche (IN) gene signature in non-obese diabetic (NOD) mice stratifies progressors from non-progressors by 6 weeks and is conserved in human T1D tissues.

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In UK Biobank patients with pulmonary hypertension (N = 248), reduced cerebellar white matter volume, thinner frontal gyri, and weaker cerebellar–frontal connectivity are linked to worse executive function and processing speed.

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Elevated TARBP2 expression in renal cell carcinoma is linked to poor prognosis and immune checkpoint blockade resistance, and it promotes immune escape by upregulating B7-H3.

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The rare TMEM175 p.L275R mutation in an early-onset Parkinson’s disease patient disrupts proton- and agonist-dependent activation gating while preserving lysosomal expression and K+ leak activity.

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In hypertensive older adults with chronic kidney disease, dapagliflozin use is linked to lower odds of progressing from prefrailty to frailty at 1 year (OR = 0.130).

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Neoadjuvant bintrafusp alfa in 6 head and neck squamous cell carcinoma patients rapidly reduces activated LRRC15+ cancer-associated fibroblasts and stromal collagen within 3 weeks.

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Preemptive rituximab-mediated B cell depletion in advanced melanoma patients receiving combined CTLA-4/PD-1 blockade is linked to fewer severe toxicities despite trial discontinuation.

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In human non-small cell lung cancer, NAT10-modified RNA accumulates in NAT10+FOXP3+ Tregs, and Nat10 loss or NAT10 inhibition reduces the PD-1+ Treg/CD8+ T cell ratio.

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Neuronal-like and secretory neuroendocrine malignant cell states were identified across 38 pancreatic and small-intestinal GEP-NETs, revealing a conserved cell-state axis.

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Nuclear cGAS binding in human cells is recruited by AP-1 transcription factors and transcriptionally regulates cholesterol homeostasis, linking lipid control to tumor growth and therapy response.

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Ruxolitinib exposure is linked to aggressive cutaneous squamous cell carcinoma (cSCC) in pharmacovigilance and a large cohort, with mechanistic support from JAK2-driven MAPK activation.

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Impaired lysosomal lipid handling in endothelial cells and microglia is linked to a neurological disorder associated with pulmonary arterial hypertension (PAH), extending cardiometabolic–neurodegenerative evidence.

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Cell-specific SMN expression and persistent peripheral immune cell alterations are demonstrated in nusinersen- or risdiplam-treated adults with spinal muscular atrophy, reframing what “adequate” SMN restoration means.

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A single liver-sparing MyoAAV4E-off AAV delivering SlugABE8e efficiently restores dystrophin expression in juvenile humanized DMD mice while reducing liver editing.

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In primary CD4+ T cells from people living with HIV-1 on ART, HIV-1–infected and uninfected cells are lysed at identical rates under CTL-engaging single-chain diabodies, with no general resistance.

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Calr-Asxl1 co-mutant mice develop more severe thrombocytosis and splenomegaly than Calr single-mutant mice, linked to a stronger megakaryocyte differentiation program.