In vivo CAR gene therapy shows promising clinical proof of concept with potential to overcome ex vivo manufacturing limitations.
Blood
Elsevier BV · Clinical Medicine · ISSN 0006-4971, 1528-0020
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Erythroid-produced intact FGF23 acts as a paracrine inhibitor of erythropoiesis, contributing to anemia in mice models of iron deficiency and chronic kidney disease.
- Open access
- 7 cites
Arlocabtagene autoleucel achieves an 87% overall response rate with durable responses in heavily pretreated relapsed/refractory multiple myeloma patients.
- Open access
- 5 cites
Ziftomenib, a potent menin-KMT2A inhibitor, induces leukemia regression and reduces tumor burden in models of KMT2A-rearranged leukemia, with strong gene downregulation.
- 6 cites
CD19 CAR T-cell therapy without lymphodepletion shows promising safety and efficacy in pemphigus vulgaris patients, with significant disease improvement in all four cases.
- Open access
- 4 cites
Mosunetuzumab plus polatuzumab vedotin achieved an 88.1% objective response rate in heavily pretreated relapsed/refractory mantle cell lymphoma patients, including high-risk groups.
- Open access
- 5 cites
Blinatumomab consolidation reduces relapse and improves survival in high-risk Ph- B-ALL patients, with 5-year relapse at 23% versus 49% in controls.
- 7 cites
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DMT1 is essential for heme-iron absorption, with knockout models showing an 8-fold reduction in uptake in rats and severe anemia in mice.
A centrally manufactured allograft therapy significantly reduces moderate-to-severe chronic GVHD and non-relapse mortality in allogeneic HSCT patients, with no increase in relapse or infection.
Menin-MLL inhibitors increase JUND activity in MLL-rearranged leukemic cells, contributing to tumor growth and therapy resistance.
- Open access
Inhibiting the mevalonate pathway reduces platelet hyperactivation and septic thrombosis, with a 50% decrease in thrombosis severity in septic mice.
Tirabrutinib monotherapy achieved a 67% overall response rate with durable effects in 48 patients with relapsed/refractory primary CNS lymphoma.
Fetal anemia and cardiac overload worsen after mid-gestation in Hb H disease, especially in Hb H-Constant Spring, with MCA-PSV peaking in late second to early third trimester.
Single-cell RNA sequencing reveals oncogenic transcriptional programs and immune escape mechanisms in over 2 million cells from CTCL patients, highlighting therapeutic vulnerabilities.
Ziftomenib combined with venetoclax and azacitidine achieves a 46% composite complete remission rate in relapsed/refractory NPM1-mutated AML patients at the 600 mg dose.
Inhibiting mitochondrial permeability transition pore (mPTP) reduces procoagulant platelet activity and thrombosis without impairing hemostasis, with a 50% reduction in thrombin generation.
Leukemia-associated myeloid cells support multiple T-ALL subtypes through distinct signals that converge on metabolic reprogramming, with AKT activation being essential for survival.
One-stage assay FVIII activity accurately reflects AAV-FVIII-SQ function and better predicts bleeding outcomes in hemophilia A gene therapy than chromogenic assays.
FGF18 from stromal fibroblasts promotes AML progression by activating FGFR3 signaling and suppressing immune responses, with elevated levels linked to poor prognosis.
- Open access
Paneth cell niche function is essential for intestinal regeneration after radiation injury but not necessary in graft-versus-host disease.
Liso-cel provides durable remission with 97% response rate and 86% overall survival at 3 years in patients with relapsed/refractory 3L+ follicular lymphoma.
Enhanced factor VIII activation by cleaving only at Arg372 improves thrombin generation and hemostatic efficacy in mice, with faster activation and lower doses needed.
Complement-mediated thrombotic microangiopathy can be effectively identified and managed despite confounding clinical triggers, with urgent complement blockade preventing irreversible kidney damage.
Functional heterogeneity of monoallelic TP53 mutations significantly impacts prognosis, with high PS and VAF predicting outcomes similar to multi-hit mutations in MDS.
Adult hematopoietic stem cells activate a fetal-restricted gene, Lin28b, to achieve functional ex vivo expansion, with overexpression improving aged HSC reconstitution by 2-3 times.
VEXAS syndrome causes endothelial activation and endotheliopathy, leading to a 52.5% thrombotic event rate and significant alterations in VWF and coagulation factors.
PCBP2 is essential for macrophage iron trafficking, with its deficiency causing iron-restricted erythropoiesis and iron accumulation in the spleen in mice.
Bone-derived DKK1 reprograms multipotent progenitor-3 cells, driving myelopoiesis and tumor progression in stage-III breast cancer.
Hematologists and oncologists can effectively lead radiation mass casualty responses using their existing skills, with 18-28% of their expertise directly applicable to managing ARS.
Venetoclax combined with inotuzumab ozogamicin achieves a 95.5% complete remission rate in relapsed/refractory B-cell ALL and LBL patients.
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