Researchers at the University of Rochester Medical Center developed a ribozyme-activated mRNA trans-ligation technique that enables the delivery of large genes for treating muscular dystrophies that require the replacement of large proteins. Muscular dystrophies are genetic disorders characterized by muscle weakness and degeneration. Treatment remains a challenge as traditional vectors, such as AAVs, are limited in their ability to carry large genetic payloads. To address this issue, researchers developed StitchR, which utilizes ribozymes to facilitate the trans-ligation of mRNA fragments. The method involves designing two mRNA fragments that can be efficiently delivered into cells using conventional vectors. Each fragment is equipped with a ribozyme that can cleave and ligate the fragments to form a full-length, functional mRNA molecule. Results demonstrated that StitchR could restore the expression of large functional muscle proteins, such as dysferlin and dystrophin, to near endogenous levels. This method opens new avenues for the efficient delivery of large therapeutic genes, potentially leading to more effective and widely applicable treatments. Congratulations to all contributors🎉! Sean Lindley Kadiam C Venkata Subbaiah Douglas M. Anderson Find out more: https://lnkd.in/e9JNkS2S #DMD #musculardystrophy #RNAtherapuetics #genetherapy #drugdelivey #geneticdiseases
Hongene Biotech Corporation’s Post
More Relevant Posts
-
2024 Nobel Prize in Physiology and Medicine: Celebrating the Discovery of miRNA! 🌟 This year’s Nobel Prize honors the breakthrough discovery of microRNAs (miRNAs)—small but mighty molecules that are reshaping our understanding of cellular control. 🧬✨ "It started in 1993 when Ambros and Ruvkun worked together as postdoctoral researchers in Massachusetts and published what they thought was an interesting finding in worms that failed to develop properly due to two mutations they identified". I personally have always been mesmerized by the crucial roles these tiny 21-24nt long single-stranded RNA molecules play as master regulators of gene expression, orchestrating the activity of more than 60% of human genes. During my PhD, I was lucky enough to delve into and work closely on miRNAs. From how precisely and conserved their biogenesis processes could be to how a minor 1-2 nt difference in their composition and sequence change the outcome of a biological pathway and results in an altered phenotype. they essentially turn a gene off at the transcriptional level, making them one of the key players in post-transcriptional gene silencing (PTGS). Their function helps fine-tune crucial processes like cell growth, differentiation, and immune responses and even reproduction in eukaryotes. RNA isn’t just a messenger; it’s one of the most important regulatory molecules in the cell, controlling gene expression and playing a pivotal role in health and disease manifestation. miRNAs, a class of that vast and diverse RNA, are emerging as game-changers in personalized medicine, offering innovative ways to diagnose and treat complex diseases like cancer, heart disease, and more. The future of gene therapy and diagnostics has never been more exciting, with miRNAs leading the way and getting the recognition they so highly deserve! 🌟🏆 #NobelPrize2024 #miRNA #RNARevolution #GeneTherapy #MolecularMedicine
To view or add a comment, sign in
-
🌟 We’re thrilled to announce a major milestone in the field of endometriosis! Scailyte, in collaboration with the University of Bern, has unveiled groundbreaking insights using our cutting-edge AI platform, ScaiVision. 🚀 🔍 Our study analyzed 466,371 cells from 60 patients, which represents the largest single-cell dataset in endometriosis to date. Using ScaiVision, we discovered key gene expression changes and altered receptor-ligand interactions in the eutopic endometrium of women with endometriosis. These findings are the of a highly specific molecular biomarker and also highlight increased inflammation, adhesion, proliferation, cell survival, and angiogenesis across various cell types. 🎯 By uncovering novel biomarkers and disease mechanisms, we’re paving the way for improved treatments in endometriosis, and most importantly, we pioneered the development of the first generation of molecular diagnostic assays in this highly prevalent and debilitating disease. 🙌 Acknowledgments We extend our heartfelt gratitude to all co-authors, study participants, physicians, nursing staff, investors, and Innosuisse for their invaluable contributions to this groundbreaking project. 📚 Read the Full Preprint https://lnkd.in/e2tBYtaT Feel free to reach out if you’d like to learn more about ScaiVision and explore how we can partner to make further groundbreaking discoveries leveraging multi-omics data and our proprietary AI platform! #singlecell #biomarkers #AI #endometriosis #womenshealth #ScaiVision
Tracing Endometriosis: Coupling deeply phenotyped, single-cell based Endometrial Differences and AI for disease pathology and prediction
biorxiv.org
To view or add a comment, sign in
-
Exciting developments in single-cell! A recent study, "Tracing Endometriosis," utilizes single-cell analysis and AI to uncover endometrial differences linked to the disease. This research enhances our understanding and improves prediction methods, paving the way for better patient outcomes. These findings highlight the transformative potential of single-cell genomics in personalized medicine, moving us closer to tailored therapies for various diseases. #SingleCellGenomics #Endometriosis #AIinHealthcare #PersonalizedMedicine
🌟 We’re thrilled to announce a major milestone in the field of endometriosis! Scailyte, in collaboration with the University of Bern, has unveiled groundbreaking insights using our cutting-edge AI platform, ScaiVision. 🚀 🔍 Our study analyzed 466,371 cells from 60 patients, which represents the largest single-cell dataset in endometriosis to date. Using ScaiVision, we discovered key gene expression changes and altered receptor-ligand interactions in the eutopic endometrium of women with endometriosis. These findings are the of a highly specific molecular biomarker and also highlight increased inflammation, adhesion, proliferation, cell survival, and angiogenesis across various cell types. 🎯 By uncovering novel biomarkers and disease mechanisms, we’re paving the way for improved treatments in endometriosis, and most importantly, we pioneered the development of the first generation of molecular diagnostic assays in this highly prevalent and debilitating disease. 🙌 Acknowledgments We extend our heartfelt gratitude to all co-authors, study participants, physicians, nursing staff, investors, and Innosuisse for their invaluable contributions to this groundbreaking project. 📚 Read the Full Preprint https://lnkd.in/e2tBYtaT Feel free to reach out if you’d like to learn more about ScaiVision and explore how we can partner to make further groundbreaking discoveries leveraging multi-omics data and our proprietary AI platform! #singlecell #biomarkers #AI #endometriosis #womenshealth #ScaiVision
Tracing Endometriosis: Coupling deeply phenotyped, single-cell based Endometrial Differences and AI for disease pathology and prediction
biorxiv.org
To view or add a comment, sign in
-
High resolution spatial profiling of kidney injury and repair using RNA hybridization-based in situ sequencing Advancements in spatial transcriptomics technologies have enabled the analysis of gene expression at cellular resolution in situ. The authors applied direct RNA hybridization-based in situ sequencing (dRNA HybISS) and developed a computational tool, CellScopes, to study gene expression in mouse kidneys, identifying cellular changes and interactions during injury and repair. https://lnkd.in/gir-qPAf
High resolution spatial profiling of kidney injury and repair using RNA hybridization-based in situ sequencing - Nature Communications
nature.com
To view or add a comment, sign in
-
It’s essential to "get a more comprehensive understanding of disease and not let patients [or] symptoms fly under the radar…" https://lnkd.in/egykPqcK Justin Moy, a panelist at this year's Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, wonderfully articulates the challenges faced in developing gene therapies for muscular dystrophies. As a PhD candidate in Bioinformatics who lives with LAMA2, Justin embodies the vital partnership between patients, scientists, and healthcare providers, offering key insights into how collaboration will inform and help progress innovation in this area. Take five minutes to watch Justin's brief, insightful interview with NeurologyLive to learn where you can make a difference in extending understanding of these diseases, documenting and sharing genotypic and phenotypic data, and establishing these crucial partnerships. Discover more details about #genetherapy (and its challenges) for Duchenne Muscular Dystrophy (#DMD) from these panel discussions: https://lnkd.in/eGAzr6PN #muscularDystrophy #patientsaspartners #raredisease
A Patient Perspective on Gene Therapy for Neuromuscular Diseases: Justin Moy
neurologylive.com
To view or add a comment, sign in
-
Our work, STew (Spatial Transcriptomic multi-viEW) to jointly model gene expression & spatial info for single-cell spatial transcriptomics data has been published at Bioinformatics Advances, Oxford University Press Read here: https://lnkd.in/gnszkU2q University of Colorado Medicine University of Colorado School of Medicine University of Colorado Department of Biomedical Informatics #DataScience #AI #Bioinformatics #Spatial
CU computational biologist Fan Zhang, PhD, and her lab have developed a new mechanism that allows researchers to combine tissue location and gene expression so that they might better learn about a disease and how to potentially target it with effective treatment. “This is really valuable because it lets physicians and researchers see not just which genes are active, but also where they are active, which can give key insights into how different cells behave and interact in diseased conditions,” Zhang explains. Read more about here: #inflammation #disease #machinelearning #softwaredevelopment
Novel Software Combines Gene Activity and Tissue Location to Decode Disease Mechanisms
news.cuanschutz.edu
To view or add a comment, sign in
-
🔬 Do you know that RNA can be present also on cell surfaces? 🧬 Recent research published in Cell reveals that cell surface RNAs (called GlycoRNAs) play a pivotal role in controlling neutrophils recruitment—an essential process in our immune response. This discovery uncovers a novel mechanism with significant therapeutic potential. Here are the main takeaways: -Cell surface RNAs regulate neutrophil movement via signaling pathways. The study reveals a previously unknown layer of complexity in cell surface biology, emphasizing the importance of surface RNAs in cellular communication. -Immune System Insight: The research provides deeper insights into how the immune system orchestrates responses to infection and injury, potentially leading to more targeted immunotherapies. -Broader Implications: The findings suggest that other surface RNAs could have similarly critical roles in various physiological processes, opening up a new frontier in cellular and molecular biology research -Therapeutic Potential: Opens avenues for targeting inflammation and immune-related diseases. -Innovation: Highlights the potential of RNA-based treatments. https://lnkd.in/djU8acfK
Cell surface RNAs control neutrophil recruitment
cell.com
To view or add a comment, sign in
-
🚀 Exciting Breakthrough in Cystic Fibrosis Treatment! 🗞️Know more information in the news article: https://bit.ly/4649Ya8 🌟 One-time prime editing is now targeting the common genetic cause of cystic fibrosis, offering new hope for patients. 🧬 This revolutionary approach could transform the way we address this condition, leading to more effective treatments and improved quality of life. 💡 Stay tuned for more updates on this groundbreaking advancement in genetic therapy! 🌐 Credit: Nature Biomedical Engineering #CFTR #DNA #CysticFibrosis #GeneEditing #MedicalInnovation #Healthcare #meded #eMednews
One-Time Prime Editing for Cystic Fibrosis - eMedNews
https://emed.news
To view or add a comment, sign in
-
Advancements in research bring in vivo CAR therapies one step closer to transformative medical breakthroughs New approach may revolutionise CAR cell production with in vivo gene editing—streamlining the process, reducing costs, and freeing scientists from labor-intensive methods. Scientists at the lab of Nobel laureate Jennifer Doudna have developed a groundbreaking method for precise in vivo genome editing using enveloped delivery vehicles (EDVs). These membrane-derived particles, cleverly cloaked in membrane fragments, facilitate the targeted delivery of CRISPR genome editing tools, such as Cas9 and gRNA proteins, to specific cells within the body. The research, published in January 2024 in the Nature Biotechnology article "In vivo human T cell engineering with enveloped delivery vehicles," demonstrates the creation of EDVs engineered at the Innovative Genomics Institute. These EDVs leverage the lentivirus's packaging ability and antibodies' cell recognition to transport molecular cargo to human cells. The study showcases successful in vivo targeting of human cells in mice with humanized immune systems, marking a significant advancement in programmable delivery modalities for potential therapeutic applications. The researchers also highlight the prospect of extending this technology to non-immune cells for targeted in vivo engineering of tissue-resident stem cells in future studies. #crispr #geneediting #cartcelltherapy #carnkcelltherapy #celltherapy #immunooncology #cancerresearch #immunotherapy https://lnkd.in/e23yXdUj
Enveloped Delivery Vehicles Enable Targeted Genome Engineering of Human Cells
genengnews.com
To view or add a comment, sign in
-
🔬 [The Shaw Prize Lecture in Life Science and Medicine 2024 – “Stepping Stones to BCL11A” and “Reactivation of Fetal Hemoglobin for Therapy”]🧬 🧪The Shaw Prize in Life Science and Medicine 2024 was awarded to two life scientists for their discovery of the genetic and molecular mechanisms underlying the fetal-to-adult hemoglobin switch, making possible a revolutionary and highly effective genome-editing therapy for sickle cell anemia and β thalassemia, devastating blood diseases that affect millions of people worldwide.🧬 In “Stepping Stones to BCL11A”, Dr Swee Lay Thein, will talk about the discovery of BCL11A gene, the key suppressor of fetal hemoglobin, and how it enabled the development of treatments for ameliorating the clinical severity of SCD and β thalassemia by inhibiting the expression of this gene to increase fetal hemoglobin. In the second part, “Reactivation of Fetal Hemoglobin for Therapy”, Professor Stuart Orkin will talk about his identification of a regulatory element (an enhancer) within the BCL11A gene itself that is required for BCL11A expression in developing red cells, and the discovery that, with gene editing tools, cutting of enhancer DNA at a single site greatly impairs BCL11A expression, thereby relieving repression of g-globin and resulting in increased HbF production. Such discovery proposed that gene editing of this site could be used to treat patients with SCD and β thalassemia, and the vision was carried forward by CrispR Therapeutics and Vertex Pharmaceuticals in clinical trials for these disorders, leading to the approval of the first gene editing therapy, Casgevy. ⚕ 📅 Date: 13 November (WED) Time: 3:00-5:00pm HKT Venue: Shaw Auditorium, HKUST 📝 Register now: https://lnkd.in/gsnvFSEy The National Institutes of Health National Heart, Lung, and Blood Institute Harvard Medical School The Hong Kong University of Science and Technology #theshawprize #shawprize #shawlaureates2024 #lifescienceandmedicine #shawlecture2024 #BCL11A
To view or add a comment, sign in
1,638 followers