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??According to Emily Mullin, in WIRED Magazine, in a groundbreaking development at the intersection of organ transplantation and genetic medicine, researchers at the University of Pittsburgh - led by Alejandro Soto-Gutiérrez - harness the power of mRNA technology to restore function in damaged livers. This innovative approach, which builds on the same mRNA technology that played a critical role in COVID-19 vaccines, aims to reprogram severely scarred livers, potentially offering a new alternative to organ transplants. ?? Collaborating with Nobel laureate Drew Weissman, the team is preparing for a clinical trial next year, marking a significant step toward revolutionizing treatments for end-stage liver disease. The potential of this research extends beyond livers. This method could be adapted by delivering specific transcription factors via mRNA to rejuvenate other vital organs, such as lungs and kidneys, impacted by chronic diseases. While the clinical application of this technology may still be on the horizon, the promise it holds for transforming the lives of patients with irreversible organ damage is immense. As the University of Pittsburgh team continues to push the boundaries of medical science, we look forward to seeing how these pioneering efforts might redefine organ regeneration and patient care. ?#mRNA #OrganTransplants https://lnkd.in/gphGA26j

??In her latest story for Nature, Mariana Lenharo explores the growing challenges in properly testing medical AI tools. Although regulators like the FDA have recently approved hundreds of AI-powered algorithms, few have undergone rigorous clinical trials. This lack of comprehensive testing raises significant concerns about the safety and effectiveness of these tools in real-world medical settings. As AI continues to revolutionize healthcare, the debate intensifies over who should be responsible for testing these technologies and how best to ensure their reliability. The current state of AI in medicine is described as a "mess" by experts. Many algorithms are approved based on limited clinical data. Hospitals and healthcare providers often find themselves in a difficult position, balancing adopting innovative tools with the need for thorough validation. As more AI tools enter the market, there is a pressing need for standardized testing and validation protocols to ensure patient safety and the efficacy of AI interventions in diverse clinical environments. The conversation around AI in healthcare is evolving, and it stresses that rigorous testing must be a priority. Read the article here: https://lnkd.in/eypxibNk #AIinHealthcare #ClinicalTrials #PatientSafety

??Researchers Mingxuan Li, Qian Fang, Pingping Xiao, Zhinang Yin, Guangbo Mei, Cheng Wang, Ying Xiang, Xuejun Zhao, Lihua Qu, Tian Xu, Jiaxi Zhang, Kejun Liu, Xiaoqing Li, Huifen Dong, Ruijing Xiao, and Rui Zhou have uncovered the critical role of KH-type splicing regulatory protein (KHSRP) in protecting against acute liver failure (ALF). ALF is a condition marked by the rapid deterioration of liver function with high mortality and morbidity rates. Through proteomic and transcriptomic analyses in murine ALF models, scientists observed the downregulation of multiple splicing factors in ALF, with KHSRP emerging as a key player. Knockdown experiments showed that the absence of KHSRP leads to significant splicing defects, such as intron retention, which exacerbates liver injury. The researchers observed KHSRP interacting directly with splicing factor SF3B1, enhancing its binding to intronic branch sites and promoting pre-mRNA splicing. These findings, published in Nature's Cell Death and Disease, suggest that KHSRP acts as a crucial splicing activator and supports the expression of genes associated with ALF progression. This discovery opens new avenues for therapeutic interventions, with KHSRP potentially serving as a target to mitigate the severity of ALF. As researchers continue to explore the molecular mechanisms underlying liver diseases, their identification of KHSRP's protective role could significantly impact future treatment strategies for ALF. https://lnkd.in/dcDkrNu5 ?#LiverHealth #ALF #MedicalResearch

????The World Health Organization (WHO) has launched a comprehensive Strategic Preparedness and Response Plan to combat the ongoing mpox outbreaks through coordinated global, regional, and national efforts. Beginning in September 2024 and running through February 2025, this plan emphasizes a multi-faceted approach, including enhanced surveillance, strategic vaccination for high-risk groups, research advancement, and community empowerment. The program's estimated cost is $135 million. WHO seeks financial support from Member States, partners like Africa CDC, and the global community to ensure effective implementation. Its goal is to stop human-to-human transmission and minimize animal-to-human transmission, ultimately controlling and halting the spread of mpox. WHO Director-General Dr. Tedros Adhanom Ghebreyesus highlighted the importance of a coordinated global response rooted in equity, solidarity, and human rights. WHO works closely with international agencies, national and local partners, civil society, and researchers to achieve this. Strategic leadership, timely evidence-based guidance, and access to medical countermeasures are at the forefront of this effort. In Africa, where the need is greatest, WHO and Africa CDC lead the charge with a unified plan and budget to spearhead the response. As the world faces this challenge, the SPRP is a vital blueprint for global cooperation and action against mpox. https://lnkd.in/g7jJEDJ9 ? #GlobalHealth #mpox #WHO

????According to Inside Precision Medicine, University College London Hospitals (UCLH) has significantly advanced cancer treatment. A lung cancer patient became the first to receive BioNTech's novel mRNA-based vaccine (BNT116). This vaccine targets cancer cells explicitly and primes the immune system to recognize and fight them, marking a new era in mRNA-based immunotherapy. The Phase I clinical trial, conducted at 34 research sites across seven countries, aims to establish the safety and efficacy of BNT116 as both a monotherapy and in combination with existing treatments for non-small cell lung cancer (NSCLC). The trial will enroll approximately 130 participants, including those with early-stage to late-stage or recurrent NSCLC. The trial represents a significant step forward in the fight against lung cancer, the leading cause of cancer deaths worldwide. By presenting common tumor markers from NSCLC to the immune system, BNT116 has the potential to enhance immune responses against cancer cells while minimizing toxicity to healthy cells. If successful, this vaccine could revolutionize cancer care by preventing the recurrence of cancer after initial treatment. The NHS is leading globally in trialing such cancer vaccines, offering new hope to patients and paving the way for future innovations in cancer treatment. https://lnkd.in/eRdSjNRm #CancerResearch #mRNA #Immunotherapy

??Hemophilia A treatment has significantly advanced, from regular factor VIII infusions to innovative gene therapies like Roctavian. While these therapies have offered new hope, they also come with limitations. For instance, patients with preexisting antibodies to the AAV capsid are ineligible for gene therapy and, if its efficacy diminishes over time, cannot be given another treatment dose. There is a considerable unmet need within the Hemophilia A community, driving ongoing research to develop more effective and accessible treatment options. One promising area of research is CRISPR/Cas9 mRNA lipid nanoparticle (LNP) gene editing, currently being explored by Dr. Carol Miao's lab at Seattle Children's Research Institute. At the ASGCT 2024 Annual Meeting, Dr. Chun-Yu Chen presented findings from their mouse model research, showcasing the potential of this approach to address some of the limitations of existing treatments. This innovative technique could offer a more durable and targeted solution for Hemophilia A patients, although further research is needed to understand its efficacy and safety fully. Such groundbreaking advancements may shape the future of Hemophilia A treatment. https://lnkd.in/ezCgS7ZZ #Hemophilia #GeneTherapy #CRISPRScience

????A recent article in Nature Communications describes how ribosome profiling has been a powerful tool for understanding the regulatory mechanisms of protein synthesis. Still, traditional methods have faced challenges, such as contamination by rRNAs and difficulty accurately measuring ribosome numbers on transcripts. To address these issues, researchers Kotaro Tomuro, Mari Mito, Hirotaka Toh, Naohiro Kawamoto, Takahito Miyake, Siu Yu A. Chow, Masao Doi, Yoshiho Ikeuchi, Yuichi Shichino and Shintaro Iwasaki have developed two innovative approaches: "Ribo-FilterOut" and "Ribo-Calibration." Ribo-FilterOut uses ultrafiltration to separate ribosome footprints from subunits, while Ribo-Calibration incorporates external spike-ins of stoichiometrically defined mRNA-ribosome complexes. Together, these methods enable a more accurate estimation of ribosome numbers on transcripts, translation initiation rates, and the total number of translation events before transcript decay, all on a genome-wide scale. This advanced ribosome profiling strategy offers more profound insights into cellular translation processes by measuring kinetic and stoichiometric parameters across the transcriptome. The method has revealed how ribosomes are allocated under various conditions, such as heat shock stress, during aging, and across different cell types. These breakthroughs in ribosome profiling enhance our understanding of protein synthesis and open new avenues for studying translation dynamics in various biological contexts. https://lnkd.in/eFWQdjcX ?#Genomics #ProteinSynthesis #MolecularBiology

??Recent research has uncovered a critical link between the enzyme oleoyl-acyl-carrier-protein (ACP) hydrolase (OLAH) and severe outcomes in respiratory infections such as avian A(H7N9) influenza, COVID-19, RSV, and multisystem inflammatory syndrome in children (MIS-C). As Cell.com reports, patients with high levels of OLAH expression early in hospitalization were more likely to experience life-threatening complications, whereas those with low OLAH expression showed better recovery outcomes. ??This discovery from researchers Katherine Kedzierska, Xiaoxiao Jia, Jeremy Chase Crawford, Deborah Gebregzabher, Zhongfang Wang, and Brendon Y. Chua highlights the role of OLAH in driving the severity of viral infections, making it a potential target for therapeutic interventions. In animal models, the absence of OLAH led to milder disease and improved survival rates during lethal influenza infections. This absence was due to reduced viral replication, less lung tissue damage, and lower inflammation levels. The research also demonstrated that supplementation with oleic acid, the main product of OLAH, increased viral replication and inflammation in macrophages. These findings suggest that targeting OLAH could be a promising strategy to mitigate the severity of viral respiratory infections and improve patient outcomes. https://lnkd.in/gM5NDuAd #RespiratoryHealth #ViralInfections #MedicalResearch

??A recent article in Nature discusses the innovative "LOOP" platform, a groundbreaking approach to inhaled mRNA therapy to treat respiratory diseases like idiopathic pulmonary fibrosis. The authors, Luke Vistain, Bijentimala Keisham, Junjie Xia, Hoang Van Phan, and Sava? Tay, describe how LOOP tackles critical challenges in pulmonary mRNA delivery, including shear force damage, mucus penetration, cellular internalization, rapid lysosomal escape, and target protein expression. Their iLNP-HP08LOOP formulation, featuring a high helper lipid ratio and specialized excipient-assisted nebulization buffer, has demonstrated exceptional stability and enhanced mRNA expression in the lungs. By delivering mRNA encoding IL-11 single chain fragment variable (scFv), this system effectively secretes IL-11 scFv in the lungs, significantly inhibiting fibrosis in preclinical models. The LOOP method outperforms both inhaled and intravenously injected IL-11 scFv in reducing fibroblast activation and extracellular matrix deposition and shows compatibility with commercially available ALC0315 LNPs. This platform represents a powerful tool for developing inhaled mRNA nanotherapeutics, opening new possibilities for treating a range of respiratory diseases. The researchers are excited about the potential of this technology to make a meaningful impact on patient outcomes, especially for those suffering from chronic and life-threatening conditions like pulmonary fibrosis. https://lnkd.in/em947RBM #mRNA #nanotechnology #pulmonaryfibrosis