Tigem

If you're in Naples, you can't miss this! 🏃♂️💙 Join the XII Walk of Life on March 23, 2025, and take a step toward hope and research for rare genetic diseases. Every stride counts! Read more here: https://lnkd.in/d7PU5Bym #WalkOfLife #Naples #FondazioneTelethon

🚀 Unlocking the Power of RNAscope in Organoids at TIGEM! 🧬🔬 We are excited to share the success of Italy’s first workshop on RNAscope in organoids and thick sections, organized by Nicolina Cristina Sorrentino, lead of the Tigem Histopathology Facility and in collaboration with our Microscopy Facility and Bio-Techne!  This hands-on training provided our researchers with cutting-edge expertise in RNA scope analysis on 3D cellular models as organoids.  RNAscope is transforming organoid-based research, offering unprecedented precision in disease modeling, drug response evaluation, biomarker discovery, and gene therapy development. By preserving the complex structure of organoids, this technology allows us to study cell-cell interactions, disease mechanisms, and therapeutic effects with exceptional accuracy.  At TIGEM, organoids are a key research focus, providing physiologically relevant models to study rare genetic diseases and evaluate treatment strategies, while also supporting the 3R principles by reducing reliance on animal models.  A special thanks to Bio-Techne, our Microscopy Facility, and all participants for making this event a success! We were also honored to welcome Mathias Van Bulck, Spatial Biology Innovation and Service Manager, EMEA, whose insights further deepened our understanding of this revolutionary technology. 🔗 Read more about this exciting initiative: https://lnkd.in/dv-TiyAP #RNAscope #Organoids #SpatialBiology #BiomedicalResearch #RareDiseases #Innovation #TIGEM 

I am excited to help organize #mSBW2025. I hope you can join me to explore latest advances in this field. Learn more at https://lnkd.in/eQF9v3UJ "

🌟New Research Alert 🌟 🧬🔍 #GeneTherapy & Liver Fibrosis – Overcoming Barriers to AAV Delivery! Liver fibrosis—a condition where scar tissue replaces healthy liver cells—can limit the success of AAV-mediated #genetransfer. Given that most #clinicaltrials exclude patients with fibrosis, understanding its impact is essential for making gene therapy accessible to more patients. 🚨 The latest study from Nature Communications, led by Pasquale Piccolo, systematically evaluated AAV-mediated liver transduction across different fibrosis models. 🔬 The study highlights that liver fibrosis: ✔️  Significantly reduces the efficiency of AAV8-mediated hepatocyte transduction ✔️ Alters vector biodistribution, redirecting AAV particles to the spleen, lung, and kidney ✔️  Affects gene transfer efficiency based on the fibrosis type and severity 🌟Importantly, the study identifies AAV-KP1 as a promising capsid variant that maintains efficient transduction even in fibrotic livers, suggesting a potential strategy for expanding gene therapy applications to fibrotic liver diseases. 🤝 This article is published alongside a study by Alessio Cantore and Chiara Simoni from San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), with both teams independently drawing comparable conclusions through distinct yet complementary model systems, further supporting the robustness of their findings. 💡 These insights pave the way for more effective gene therapies, ensuring better treatment outcomes for patients with fibrosis-linked diseases. 🔗 Read more: https://lnkd.in/dGwa3yaY #LiverFibrosis #AAV #GeneTherapy

🔬 Exciting news from the Advanced Histopathology Facility! We are thrilled to partner with Bio-Techne to host the 1st Italian Workshop on RNAscope in Organoids and Thick Sections! 🧬 📍 Location: TIGEM, Via Campi Flegrei 34, Pozzuoli 📅 Date: March 11-13 This hands-on workshop is a unique opportunity to explore cutting-edge RNAscope technology with leading experts in the field. If you're working with organoids or thick tissue sections, this is an event you won’t want to miss! #RNAscope #Organoids #Histopathology #Innovation #Workshop #TIGEM #BioTechne Nicolina Cristina Sorrentino Euplio Visco Mathias Van Bulck

🌍 #RareDiseaseDay On this Rare Disease Day, we recognize the strength, resilience, and remarkable journeys of those living with rare diseases, along with their families and caregivers, as we reaffirm our commitment to collaboration, advocacy, and empowerment. 🤝 Together with our partners and the rare disease community, we work to expand knowledge, drive innovation, and develop solutions that bring hope and healing. 🍀 Although each rare disease affects only a small percentage of the population, together they impact millions worldwide. At Tigem, we recognize the unique challenges faced by those living with rare diseases and remain committed to advancing research, deepening understanding, and providing support for these communities. Take part in celebrating Rare Disease Day and showing support for those whose lives are affected by rare diseases. United, we can create change. #rarediseases #fondazionetelethon

🌸 #InternationalWomensDay 🌸 March 8th is not just a celebration, but an opportunity to reflect on the fundamental role of women in every aspect of society. Women are the driving force of change: they lead, innovate, educate, and inspire. They are scientists, leaders, entrepreneurs, mothers, educators, and tireless workers. Every day, with talent and determination, they break down barriers and help build a more equitable future. In the fields of research, politics, economics, art, and technology, women continue to leave an indelible mark. Today, and every day, we celebrate the strength, resilience, and talent of women. Because without them, the world would simply not be the same. 💜 Happy International Women's Day! 💜 #InternationalWomensDay #March8th

🌟 We are excited to share a new study published in #TheJournalofPhysiology by Galietta’s group, which uncovers how the airway’s mucus layer significantly alters the effectiveness of CFTR-targeting drugs—a key factor in cystic fibrosis (CF) research. The findings suggest that mucus acts as a barrier, limiting drug access and reducing pharmacological response 📚 Key Findings from the Study Researchers observed that CFTR inhibitors, such as CFTRinh-172 and PPQ-102, only partially blocked chloride transport in human airway epithelial models. However, removing mucus with a reducing agent restored full drug efficacy, revealing the mucus layer’s critical role in drug permeability. 🌟 Implications for Cystic Fibrosis Treatment These results highlight the need to consider mucus composition when assessing CF therapies. Enhancing drug penetration through mucus removal or modification could improve treatment outcomes, paving the way for more effective CFTR-targeting drugs. 📖 Read the full article here: https://lnkd.in/dUjNsUjD #CysticFibrosis #MucusBarrier #CFTR #DrugDiscovery #AirwayResearch

🌟New Research Alert 🌟 🔬 We are thrilled to share a groundbreaking study by De Matteis’s group, published in #LifeScienceAlliance, uncovers a crucial link between mitochondrial dysfunction and protein aggregation in Amyotrophic Lateral Sclerosis type 8 (ALS8). Using a yeast model and mammalian motoneuron cells, the researchers demonstrate how mutant VAPB—a key ER protein—forms toxic aggregates via ER-mitochondrial contact sites, driving ALS8 progression. 📚 Key Findings from the Study ALS8 is caused by a P56S mutation in VAPB, leading to misfolded protein aggregates in neurons. The study shows that these aggregates form in response to mitochondrial dysfunction and are exacerbated by ER-mitochondrial interactions. Notably, reducing these contacts prevents aggregate formation, identifying a potential therapeutic target. 🌟 A New Therapeutic Avenue By uncovering the role of ER-mitochondrial crosstalk in ALS8, this research highlights a novel strategy to slow disease progression. Targeting these inter-organelle interactions could pave the way for new ALS therapies, offering hope for patients with this devastating neurodegenerative disorder. 📖 Read the full article here: https://lnkd.in/dcfc4RJK #ALS #Neurodegeneration #Mitochondria #ERstress #CellBiology

Amazing team work Giulia Maule Irene Carrozzo Carmelo Gentile Matteo Ciciani Alessandro Umbach and collaborators Luis Galietta group Tigem. This work shows how secondary non pathogenic mutations can be leveraged as therapeutic strategies to neutralize the main CF causing mutation. #cystic fibrosis #CRISPR #FFCricerca https://lnkd.in/dhTS6PYH