Genethon reported positive results from its AAV-microdystrophin treatment trial in Europe. The three participants receiving the study’s higher dose (3x1013vg/kg) showed that an average of 54% of muscle fibers were producing microdystrophin, and individuals demonstrated a reduced level of creatine kinase, a marker of muscle damage. Genethon said it is planning a pivotal trial in Europe. #CureDuchenne #duchenne #DMD #wewillcureduchenne
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According to the newest recommendations released by the NCCN testing of ROS1 rearrangements is required prior to the use of TKI inhibitors, as it is in ALK positive patients. Crizotinib used in metastatic and advanced NSCLC significantly improves the condition. Note : break apart probe: f 1 rearrangement of ROS1 gene f-2 normal signal pattern. DIAGNOSED WITH NSCLC. #solid #tumors #cytogenetics #patient #safety #protocols
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Organoid vs. Spheroid in 3D Cell Cultures Organoids, the intricate replicas of specific organs, excel in mimicking both the structure and function of human tissues. They offer invaluable insights into disease mechanisms and potential treatments. On the other hand, spheroids, though simpler, provide a versatile platform for studying various cellular behaviours, including drug responses and cancer microenvironments. Share your insights in the comments and be part of the discussion. #CellCulture #OrganoidVsSpheroid #MedicalResearch #StemCells #HealthcareInnovation #ResearchInsights #Biotechnology #LabLife #ScienceDiscussion #AskTheExpert #ResearchDebate
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Antisense oligonucleotides (ASOs) are promising tools in drug development that can be used to precisely control gene expression and target a range of diseases at the molecular level. Our scientists, Amy Byrnes, Casper Hoogenraad and team published in Cell Reports Methods about the development of a new pre-clinical model that emits green fluorescence in tissues when ASOs are active. This is a critical step forward in understanding how ASO activity is regulated in the body, which is important for developing ASO therapies for a wide-range of diseases. Learn more about their work: http://spr.ly/6049XUzqN Imaged is a mouse's brain, focusing on two regions known as the hippocampus and cortex. In this photo, you can observe these areas fluorescing green after injection with ASOs, shown in red.
A fluorescent splice-switching mouse model enables high-throughput, sensitive quantification of antisense oligonucleotide delivery and activity
sciencedirect.com
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Paper of the week Pendse et al recently showed an important role of C1q produced by macrophages in regulation of gastrointestinal motility. Macrophages were shown to be the predominant source of C1q in the mouse intestine and most extraintestinal tissues. C1q expressing macrophages were located in the intestinal submucosal myenteric plexuses and closely associated with enteric neurons. This C1q was not essential for immune defense of the intestine. Mice with macrophage-specific deletion of C1qa exhibited an altered enteric neuronal gene expression, as well as increased neurogenic activity of peristalsis, and accelerated intestinal transit. Check out their work here: https://lnkd.in/gy3FFgCF
Macrophages regulate gastrointestinal motility through complement component 1q
elifesciences.org
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Using #CRISPR/#Cas13a system, researchers develop easy-to-use method that can detect small amounts of #cancer-related molecules in #exosomes in plasma distinguishing between malignant & benign samples. bit.ly/3wJLBxV #biomarkers #microRNA #cancerdiagnostics #breastcancer
Highly Effective Detection of Exosomal miRNAs in Plasma Using Liposome-Mediated Transfection CRISPR/Cas13a
pubs.acs.org
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Explore the five critical steps in our research and development process to advance next-gen therapeutics for rare neuromuscular diseases. Together, we're rewriting the future of medicine. Learn more here: https://loom.ly/3zOEXR4 #CureRareDisease #NeuromuscularDiseases #CRD #Process
Our Process | Cure Rare Disease
cureraredisease.org
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Combination therapy to treat glioma In this study, they used treatment-naïve patient-derived DIPG cell line and performed epigenetic compounds screening to identify that gemcitabine as a candidate. As a pyrimidine analog, gemcitabine stabilized and activated p53, including increasing chromatin accessibility for p53 at apoptosis-related loci. Notably, the H3.3K27M mutation facilitates gemcitabine-induced apoptosis in DIPG. However, gemcitabine induced the transcription of ERV (endogenous retrovirus), which might activate cGAS-STING signaling, and stimulated non-canonical NFκB signaling. A drug screen in gemcitabine-treated DIPG cells revealed that fimepinostat, a dual inhibitor of HDAC and PI3K, effectively suppressed the gemcitabine-induced NFκB signaling in addition to blocking PI3K/AKT activation. Combination therapy comprising gemcitabine and fimepinostat elicited synergistic anti-tumor effects in vitro and in orthotopic H3.3K27M DIPG xenograft models. #sciencenewshighlights #ScienceMission https://lnkd.in/gXEDPKK2
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It’s important to quantitate cytokines and chemokines specific to #neuroinflammation in multiple sample types, and MILLIPLEX® multiplex and SMC® ultrasensitive assays can meet your research needs, whether assaying multiple protein biomarkers or requiring fg/mL detection.
Neuroimmunology & Neuroinflammation Immunoassays
sigmaaldrich.com
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Good review of different pathways to cell death, and consequences of each type of death.
Very nice review that covers cell death pathways and touches upon dead cell elimination. Understanding the consequences of a cell death pathway in terms of the type of debris generated and the mechanisms for clearing that debris (ex. inflammatory vs non-inflammatory) is as important to drug discovery as understanding the cell death pathway itself. Ideally all three elements, cell death pathway, debris and clearance mechanisms, should be incorporated into a biomarker strategy that is indicative of drug action independent of efficacy in early development to know if, when and how to progress treatment.
A guide to cell death pathways - Nature Reviews Molecular Cell Biology
nature.com
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Very nice review that covers cell death pathways and touches upon dead cell elimination. Understanding the consequences of a cell death pathway in terms of the type of debris generated and the mechanisms for clearing that debris (ex. inflammatory vs non-inflammatory) is as important to drug discovery as understanding the cell death pathway itself. Ideally all three elements, cell death pathway, debris and clearance mechanisms, should be incorporated into a biomarker strategy that is indicative of drug action independent of efficacy in early development to know if, when and how to progress treatment.
A guide to cell death pathways - Nature Reviews Molecular Cell Biology
nature.com
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