SNP-guided epigenetic repression of mutant alleles can induce compensatory expression from non-targeted alleles, maintaining gene dosage in dominant genetic disorders.
Cell Stem Cell
Elsevier BV · Cell & Molecular Biology · ISSN 1934-5909, 1875-9777
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Phagocytosis-shielded BaEVRLess-pseudotyped vectors achieve up to 8.8% gene marking in human HSCs in vivo, enabling potential gene therapy for sickle cell disease.
Transformer base editing of HBG1/2 promoters shows promising responses across diverse β-hemoglobinopathies, including sickle cell disease and various β-thalassemia genotypes.
Clinical base editing achieves transfusion independence and durable fetal hemoglobin expression in diverse β-hemoglobinopathy patients, including an African SCD case, with no adverse effects.
Endothelialized callus organoids enable rapid regeneration of large long bone defects in mice, achieving functional repair within a short timeframe.
Epi-Allele epigenetic editing induces compensatory non-targeted allele expression, preventing haploinsufficiency in dominant genetic diseases, with potential for broad therapeutic use.
ASO-tagged siRNA successfully delivers SOD1 gene silencing to the human CNS, achieving improved targeting in ALS patients with enhanced tolerability.
Antibody-engineered lipid nanoparticles enable efficient, durable in vivo editing of human hematopoietic stem cells, advancing gene therapy for blood disorders.
Activin B-ACVR2A signaling drives growth in diffuse midline gliomas, with a new patient-derived organoid model revealing a promising triple intrathecal therapy.
Hippo signaling controls Igf2 expression to regulate liver growth and regeneration, highlighting a key mechanism for organ size calibration in postnatal stages.
Immunoids integrating immune cells into human pluripotent stem cell-derived organoids reach advanced development stages, promising to revolutionize disease modeling and therapy.
Genomic and transcriptomic qualification of patient-derived dopaminergic neuron precursors was successfully achieved across multiple donors for Parkinson's disease therapy.
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H3K27M-mutant brainstem glioma organoids reveal OPC stemness driven by dysregulated ACTIVIN signaling and identify effective intrathecal therapy.
Hippo-IGF2 signaling axis controls organ size by regulating growth during development and regeneration, with Igf2 restoring liver growth in aged mice.
PD-L1/2-null human iPSC-derived dendritic cells dressed with tumor MHC complexes activate potent anti-tumor T cell responses, demonstrating a scalable vaccine platform.
Engineered mesenchymal stem cells convert tumor stiffness into a detectable and targetable marker, enabling PET imaging and radionuclide therapy in previously untargetable tumors.
Complement-driven stromal reprogramming in the spleen promotes myelofibrosis, with C3 deficiency or inhibition reducing fibrosis and splenomegaly in mice.
Organized pathological hematopoiesis occurs in the myelofibrotic spleen, involving complex cellular and stromal remodeling, challenging the idea of simple compensation.
Splenic extramedullary hematopoiesis in myelofibrosis involves significant transcriptomic and epigenetic dysregulation, with expansion of progenitor cells and immune microenvironment remodeling.
Cholesterol buildup in APOE4 astrocytes drives α-synuclein aggregation, increasing pathology by 50% in human brain-like tissue models.
Bioengineered human heart valve-like tissues from pluripotent stem cells mimic native valves and reveal key features of valve maturation and disease.
TIGRa, a compact and multiplexable gene activator, achieves efficient activation of up to 12 genes and reprograms human cells with less than half the size of traditional systems.
Short-term morphogen exposure creates neocortical organoids with lasting anteroposterior patterning, matching prenatal human cortex signatures and enabling disease modeling.
Engineered MSCs detect tumor stiffness to enable targeted radiotheranostics, achieving sensitive detection and treatment across multiple tumor models.
Ovarian aging drives systemic aging through cellular defects and hormonal decline, with interventions showing potential to extend healthspan and preserve fertility.
Human iPSC-derived valve-like assembloids replicate key features of human valve development and disease, providing a new platform for in vitro studies.
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