Scientists at the University of Utah have achieved something groundbreaking—new gene therapy has successfully reversed heart failure in a large animal study. Jasdeep Dalawari MD FACC FSCAI FSVM, Regional Chief Medical Officer at VitalSolution, weighed in on these exciting findings, saying, "Gene therapy, precision medicine, and personalized health care is the future." Dr. Dalawari also highlighted the bigger picture, noting, "There are many genetic modifications happening in different diseases, like cystic fibrosis and muscular dystrophy, that are looking at a similar intervention – injecting healthy genes in the hopes of finding cures." This study showed a 30% improvement in heart function, a massive leap from the 5-10% improvements seen in previous efforts. Learn more about the study: https://lnkd.in/gU6XCGCE
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I am pleased to announce the successful completion of our group presentation on applying adenoviral gene therapy in cystic fibrosis, as part of our Human Physiology and Mechanism of Diseases course. Our comprehensive analysis covered the recent advancements in adenoviral vector technology and its potential to improve therapeutic outcomes for cystic fibrosis patients significantly. We discussed the latest research findings, current challenges, and the promising future directions in this innovative field. I am grateful for the opportunity to collaborate with such dedicated and insightful colleagues. Together, we have deepened our understanding of how gene therapy can revolutionize the treatment landscape for cystic fibrosis and beyond. #GeneTherapy #CysticFibrosis #MedicalResearch #Innovation #HumanPhysiology #FutureOfMedicine #CollaborativeLearning #Biotechnology #Healthcare #ScientificResearch #MedicalAdvancements #Genetics #AdenoviralVectors #TeamScience #Teamwork
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Hot off the presses! Are you utilizing minipigs in your AAV gene therapy research, or have you considered them as your larger animal model? Scientists at VRL Laboratories recently published in Viruses. Read more to learn why the Göttingen minipig might be a great choice for your next study. Prevalence of Antibodies against Adeno-Associated Viruses (AAVs) in Göttingen Minipigs and Its Implications for Gene Therapy and Xenotransplantation https://lnkd.in/g4KvFcFe
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I'm pleased to share our recently published study, where we conducted an extensive analysis of whole genome sequencing (WGS) datasets to understand the impact of gene editors. 🧬 Our research evaluated the use of adeno-associated virus (AAV) vectors for gene editing in neonatal mice livers. The results are promising: targeting the factor IX gene (F9) proved to be highly efficient, and off-target effects were extremely rare. 🔬🚀 Curious to learn more? Check out the full article: https://lnkd.in/gw2FAFze 👏 🧪 🌟 Innovate & Explore! 🌟 #GeneEditing #Biotech #Research #CRISPR #Genomics #ScienceInnovation #AAVVectors #BiomedicalResearch
Comprehensive Analysis Of Off-Target And On-Target Effects Resulting From Liver-Directed Crispr-Cas9–Mediated Gene Targeting With Adeno-Associated Viral Vectors
cell.com
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Retina Australia is delighted to announce the first of two $60,000 2025 Research Grant recipients, Professor Robyn Jamieson from the Children's Medical Research Institute, Sydney Children’s Hospitals Network, and Save Sight Institute - University of Sydney. Robyn's project aims to develop gene replacement therapies for autosomal recessive (AR) inherited retinal diseases (IRDs), where no treatments currently exist. It focuses on targeting genes small enough for adeno-associated virus (AAV)-mediated gene therapy, which has shown promise in other successes like RPE65 gene therapy. For more information about the life changing research funded by Retina Australia, visit https://tr.ee/QziF-0ICmu #retinaaustralia #inheritedretinaldisease #eyeresearch
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#ARVO2024 join us for a lighting session tomorrow on how I developed multiple therapies to treat rare and common retinal diseases! And learn how #ShifaPrecision will use precision medicine to identify the most effective personalized therapies!
We're at #ARVO2024 in Seattle! Come see us at booth 1143, and join us for a rapid-fire research showcase on Tuesday, May 7, at noon, featuring seven AMDF supported researchers working in the macular degeneration space. Our headliner is Neena Haider, Ph.D., presenting: Modifier Gene Therapy for AMD, Stargardt's and Retinitis Pigmentosa. "A mutation/gene-agnostic modifier gene therapy approach that restores homeostasis, halting disease progression along multiple pathways and rescuing dying photoreceptors." #maculardegeneration #stargardtsdisease #retinitispigmentosa #genetherapy #eyeresearch #ARVO Image: Dr. Haider in front of a microscope, looking into the camera, holding a microscope slide. In the background is a lab.
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Hot Topic of the Week: Bridge RNAs This week, we will focus on the research advances of Bridge RNAs (bRNAs) in five key areas. (1) Functional Roles: understanding of bRNAs as a key mediator of gene regulation and cellular communication. (2) Technological Progress: high-throughput sequencing has greatly enhanced the study of bRNAs, allowing for detailed exploration of their interactions. (3) Therapeutic Potential: highlighting the applications of bRNAs in gene therapy, with ongoing research aimed at unlocking their potential in treating genetic diseases. (4) Regulatory Mechanisms: investigating how bRNAs participate in complex regulatory networks that influence a variety of biological processes. (5) Future Directions: exploring the need to continue exploring their roles in disease and their potential as therapeutic targets.
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Scientists at the University of Utah have achieved something groundbreaking—new gene therapy has successfully reversed heart failure in a large animal study. Dr. Jasdeep Dalawari, Regional Chief Medical Officer at VitalSolution, weighed in on these findings, saying, "Gene therapy, precision medicine, and personalized health care is the future." This study showed a 30% improvement in heart function, a massive leap from the 5-10% improvements seen in previous efforts. Imagine the potential lives this could impact as research progresses into human trials! Learn more about the study: https://lnkd.in/gZAPCnmN
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First report of an anellovirus-based #gene_therapy vector, with preclinical data demonstrating durable in vivo function in the mouse eye for 9 months after subretinal administration. Human #anelloviruses are commensal/non-pathogenic, evade induction of humoral immune responses, have enormous genomic diversity and wide tissue distribution, and may offer a safe/redosable alternative to other viral delivery platforms used in gene therapy. https://lnkd.in/e2FDkqMP
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Exciting news in the biomedical field! Researchers have identified key genetic factors contributing to Inflammatory Bowel Disease (IBD). In a study published in the Proceedings of the National Academy of Sciences, researchers detailed how histone deacetylases (HDAC4 and HDAC7) function distinctively yet cooperatively in regulating ordered gene transcription during Th17 cell differentiation, providing new insights into inflammatory processes. This breakthrough sheds light on the genetic regulation of inflammation, significantly advancing our understanding of diseases like Crohn’s disease and ulcerative colitis. The discovery holds promise for developing targeted therapies. #IBDresearch #BiomedicalResearch #Genetics #IBD #CrohnsDisease #UlcerativeColitis #Inflammation #Th17Cells #HDAC #GeneticRegulation #TargetedTherapies #FutureofMedicine
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𝗛𝗲𝗮𝗿𝘁 𝗳𝗮𝗶𝗹𝘂𝗿𝗲 𝗺𝗮𝘆 𝘀𝗼𝗼𝗻 𝗵𝗮𝘃𝗲 𝗮 𝗻𝗲𝘄 𝗰𝘂𝗿𝗲 Researchers at the University of Utah College of Health have developed a cutting-edge gene therapy capable of reversing heart failure in a large animal model with just a single IV injection. By using an adeno-associated virus (AAV9) to deliver the cBIN1 gene directly into heart cells, this therapy replenishes a crucial protein often depleted in heart failure. This innovative approach offers hope for millions affected by cardiac diseases, potentially transforming how we treat and manage heart failure in the future. Science is truly paving the way for a healthier tomorrow! What do you think? #HealthcareInnovation #GeneTherapy #Cardiology #MedicalResearch #Researcher #Work #Progress #Potential
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