Beti-cel Gene Therapy Sustains Transfusion Independence for Up to Ten Years
Long-term data show 90.2% of phase 3 patients achieved transfusion independence with stable hemoglobin and improved iron homeostasis.
POT1 Mutations Drive Lymphoid Malignancy Through Ultralong Telomeres
Pathogenic POT1 variants increase lymphoma risk eightfold by extending cellular longevity and promoting age-dependent clonal expansion.
Ruxolitinib Resolves Severe Diarrhea Following BCMA-Targeted CAR T-Cell Therapy
A case series suggests JAK inhibition may mitigate the high mortality associated with immune-mediated enterocolitis in multiple myeloma.
NXT007 Reaches Nonhemophilic Factor VIII Levels in Severe Hemophilia A
A phase 1/2 trial shows monthly subcutaneous NXT007 achieves factor activity above 40 IU/dL and eliminates bleeds at higher doses.
Interleukin-4 Inhibits Platelet Glycoprotein VI to Reduce Thrombosis in Mice
The anti-inflammatory cytokine IL-4 binds directly to the GPVI receptor, suppressing collagen-induced activation and clot formation.
Integrated Genetics and MRD Guide Transplant in Ph-Negative ALL
A prospective study shows that combining genetic risk with early molecular response identifies adults who can safely avoid transplantation.
RNA Interference Normalizes PAI-1 Levels and Prolongs Lifespan in Murine Models
A single dose of siRNA-lipid nanoparticles achieved 90% knockdown of plasma PAI-1, reducing thrombus weight and increasing survival in mice.
Triplet Immunotherapy Shows Durable Response in Relapsed Hodgkin Lymphoma
Adding ipilimumab to nivolumab and brentuximab vedotin may improve long-term disease control for patients deferring stem cell transplant.
Immune Score Predicts Infection Risk in Multiple Myeloma
A three-factor immune profile identifies patients with a 60% infection incidence, offering a path to personalized prophylaxis.
Targeting Donor Dendritic Cell Subsets Prevents Lethal Gut Graft-Versus-Host Disease in Mice
Researchers identified XCR1+ and CD11b+ dendritic cells as drivers of Th1 and Th17-mediated intestinal damage after transplantation.
Plasmodium knowlesi Adapts In Vitro to Infect Duffy-Negative Erythrocytes
A chimeric gene allows the zoonotic parasite to bypass the Duffy-antigen receptor, suggesting potential for wider geographic spread.
ANKRD26 Overexpression Triggers Megakaryocyte Apoptosis via JUNB-p21 Pathway
Single-cell analysis of bone marrow reveals that elevated ANKRD26 at the centrosome causes premature death of polyploid platelet precursors.