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VectorY Therapeutics Announces First Participant Dosed in Phase 1/2 PIONEER-ALS Clinical Trial of VTx-002 in People with Amyotrophic Lateral Sclerosis (ALS)
[VectorY Therapeutics] VectorY Therapeutics announced that the first participant has been dosed in its Phase 1/2 PIONEER-ALS trial evaluating VTx-002, a first-in-class vectorized antibody targeting TDP-43 pathology in people with ALS.
Glucosylceramide-Induced Ectosomes Propagate Pathogenic α-synuclein in Parkinson’s Disease
[Nature Cell Biology] Investigators used live-cell microscopy to examine the impact of Parkinson’s disease associated lipid alterations on α-synuclein release. They discovered that increased glucosylceramides as a consequence of reduced β-glucocerebrosidase activity induce ectosome shedding from primary neurons.
HSPA5 Promotes YAP/TAZ Stability Independently of the Hippo Pathway and Induces Proneural-to-Mesenchymal Transition in Glioblastoma
[Cell Death & Disease] Scientists demonstrated that heat shock protein family A member 5 (HSPA5) expression was highest in the mesenchymal subtype of glioblastoma. The overexpression of HSPA5 in proneural cells induced proneural-to-mesenchymal transition and promoted malignant phenotypes.
NeuroD1-USP1-MYCN Axis Drives Tumor Progression in Neuroblastoma
[Journal of Translational Medicine] The authors investigated the upstream regulatory mechanisms of N-Myc stabilization in neuroblastoma and explored potential therapeutic strategies targeting these mechanisms.
Targeting of HSP27 and MMP-2/9 Crosstalk by High-Throughput Drug Repurposing Strategies Identifies Paroxetine As a Potential Candidate in Glioblastoma
[Journal of Medicinal Chemistry] Investigators established paroxetine as a functional heat shock protein 27 (HSP27) inhibitor that disrupts the interaction between HSP27 and MMP-2/9, thereby inhibiting glioblastoma progression.
Wnt-3a Exacerbates Production of TNF-α in LPS Stimulated Microglia Independent of the β-Catenin Canonical Pathway
[Scientific Reports] Researchers found that Wnt-3a alone had no effect on pro-inflammatory TNF-α or IL-1β release from homeostatic primary microglia, however co-administration with LPS significantly increased TNF-α release beyond that seen with LPS alone.
APP-C31 Pathology As a Target in Neurodegenerative Diseases
[Journal of Biomedical Science] Scientists provide a comprehensive introduction to the structure, neurotoxicity, regulatory mechanism, and relevance of APP-C31 to clinical diseases and its therapeutic potential as a drug target.
Microglia-Associated Progression of Multiple Sclerosis: Target Identification and Therapeutic Engagement in Human In Vitro Models
[Experimental & Molecular Medicine] Investigators highlight advances in modeling MS progression, using human induced pluripotent stem cell-derived systems, with a particular focus on microglia as key mediators of neuroinflammation and neurodegeneration.
Targeting Metabolic Mechanisms to Overcome Temozolomide Resistance in Glioblastoma
[Discover Oncology] The authors summarize resistance-associated changes across three key areas – glycolysis, redox homeostasis, and lipid metabolism in temozolomide-resistant glioblastoma.
Amide-Forming Ligations at Physiological Ph for the Encapsulation of Human Mesenchymal Stem Cells
[Biomacromolecules] Investigators demonstrated the construction of poly(ethylene glycol)-derived hydrogels by efficient cross-linking of quinolinium acyltrifluoroborate-functionalized macromers with a partner hydroxylamine-functionalized macromer.
MSCs Delivering LIGHT Prime Immune Response against CAFs to Harness Antigen Loss Variants
[Cancer Gene Therapy] The authors engineered MSCs to deliver the immuno-stimulatory TNF superfamily ligand LIGHT (MSC-L). They found that MSC-L simultaneously primed immune responses against both tumor cells and cancer-associated fibroblasts.
Human Bone Marrow Derived Mesenchymal Stem Cells Do Not Promote Oral Cancer Cell Growth In Vitro and Metastasis In Vivo
[Scientific Reports] Scientists assessed the effect of circulatory bone marrow derived-MSCs on proliferation, migration, invasion, tumor growth, and metastasis of oral squamous cell carcinoma cells.

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