The Shaw Prize’s Post

💎💎💎Whatsapp!! 💎💎💎First Spliceosome Blueprint Reveals Complex Interplay, Uncovers Drug Targets Researchers at the Centre for Genomic Regulation (CRG) have created the first #blueprint of the human #spliceosome, the #complex #molecular #machine that edits genetic messages transcribed from DNA, allowing cells to create different #protein versions from a single gene. Their results could lead to new #therapeutic approaches that target #RNA processing in diseases, such as some cancers, that are linked to faulty RNA molecules produced by mistakes in #splicing. The detailed map of the #spliceosome, which the authors of the study have made publicly available, can help researchers pinpoint where the splicing errors occur in a patient’s cells. Creating the resource took more than a decade. “We wanted this to be a valuable resource for the research community,” said Institució Catalana de Recerca i Estudis Avançats (ICREA) research professor Juan Valcárcel, PhD, a researcher at the CRG. “#Drugs correcting splicing errors have revolutionized the treatment of rare disorders like spinal muscular atrophy. This blueprint can extend that success to other diseases and bring these treatments into the mainstream.” Added Malgorzata Rogalska, PhD, co-corresponding author at the CRG, “Current splicing treatments are focused on rare diseases, but they are just the tip of the iceberg. We are moving into an era where we can address diseases at the transcriptional level, creating #disease_modifying #drugs rather than merely tackling symptoms. The blueprint we’ve developed paves the way for entirely new therapeutic approaches. It’s only a matter of time.” Valcárcel is the senior author of the scientists’ report in Science, “Transcriptome-wide splicing network reveals specialized regulatory functions of the core spliceosome,” in which they concluded, “Given the prevalence and functional relevance of the splicing process and its regulation, the resources, approaches, and insights provided in this and related studies have the potential to illuminate underlying physiological and #pathological #mechanisms and eventually inform the design of new therapeutic approaches. ➡ 💎 You can find more pieces of work by clicking here. https://lnkd.in/eSG67K5G #disease_modifying #drugs #spliceosome

🔬 CRISPR-Cas9: Transforming the Future of Personalized Medicine 🔬 Advances in gene-editing technologies, particularly CRISPR-Cas9, are ushering in a new era of precision medicine, where treatments are tailored to the unique genetic makeup of individuals. CRISPR-Cas9 enables scientists to precisely modify DNA, providing unprecedented opportunities to: Correct genetic mutations underlying rare and inherited diseases. Enhance immunotherapies, such as CAR-T cells, to better target and eliminate cancer cells. Develop tailored gene therapies that address the molecular drivers of complex diseases. In personalized medicine, CRISPR-Cas9 is a game-changer, allowing us to move beyond one-size-fits-all treatments to interventions designed for specific patients. For instance, by integrating CRISPR with cutting-edge tools like RNA sequencing and single-cell analysis, we can gain deeper insights into individual disease mechanisms and response to therapies. While the promise of CRISPR-Cas9 is extraordinary, it also demands careful ethical considerations and rigorous oversight to ensure safety and equitable access. As we embrace this powerful technology, collaborative efforts between researchers, clinicians, and policymakers will be essential. The potential to redefine healthcare is within reach, and the role of CRISPR-Cas9 in personalized medicine will undoubtedly be pivotal. What do you envision as the most impactful applications of CRISPR in precision medicine? Let’s discuss! #CRISPR #PrecisionMedicine #GeneEditing #PersonalizedHealthcare #PersonalizedMedicineJournal #AdvancedTherapiesJournal #AmitisGen

Exciting News in Transplant Medicine! Verici Dx is excited to share the latest advancements in post-kidney transplant care, where RNA sequencing technology coupled with machine learning is revolutionizing our approach to differentiating and managing complications like BK virus nephropathy (BKN) and acute rejection. RNA sequencing enables a comprehensive analysis of gene expression patterns, offering valuable insights into the molecular mechanisms underlying these conditions. By analyzing gene expression profiles, researchers are identifying unique biomarkers specific to rejection enabling clinicians to differentiate between BKN and acute rejection, paving the way for more precise diagnosis and personalized treatment strategies. One such promising biomarker is Tutivia, a leading-edge diagnostic tool that utilizes RNA sequencing technology to analyze gene expression patterns associated with acute rejection. Tutivia offers a non-invasive and highly specific method for risk probability of acute rejection with no false positives occurring in patients with BK nephropathy, helping clinicians tailor treatment plans to each patient's specific needs. With Tutivia and RNA sequencing technology, we're not just identifying injury that has occurred – we're getting to the root of the problem, offering actionable data, and advancing the field of transplant medicine. It's an exciting time for innovation and progress in healthcare, and Verici Dx is thrilled to be a part of it! https://lnkd.in/gjQ-NNQw #TransplantMedicine #RNASequencing #ClarityEvenEarly #PrecisionMedicine #TutiviaDelivers #HealthcareInnovation

🌟 Excited to Share Our Latest Publication! 🌟 Happy to announce that our review article has been published in the Life Sciences journal, titled "Personalized Medicine in Cystic Fibrosis: Exploring Genetic Foundations and Precision Therapeutics." This review explores into the transformative role of personalized medicine in cystic fibrosis (CF) treatment, highlighting: ✅ Genetic Foundations: Exploring the impact of CFTR gene mutations and their correlation with disease severity. ✅ CFTR Modulators: Advances with Ivacaftor, Lumacaftor, and Tezacaftor, tailored to individual genetic profiles. ✅ Emerging Therapies: The potential of gene therapy, genetic manipulation, and CRISPR-Cas9 for mutation correction. ✅ Biomarkers: Novel inflammatory markers and omics technologies for early detection and disease progression monitoring. ✅ Pharmacogenomics: Personalized approaches to optimize drug efficacy and minimize adverse effects. We also address the ethical and regulatory challenges surrounding equitable access and long-term sustainability of these groundbreaking therapies. As CF care transitions towards precision medicine, we hope this article inspires further research and innovation in the field. 🌐 I thank and congratulate my coauthors Dr. Meghana G S and Dr. Akhila Akkihebbal Ravikumar and Good work Abinesh R S You can read the full article here: https://lnkd.in/ddGAPduH Looking forward to engaging discussions and collaborations in the area of personalized medicine and CF treatment! 🚀 #CysticFibrosis #PersonalizedMedicine #PrecisionTherapies #GeneTherapy #Pharmacogenomics #LifeSciences #Research #Elsevier

* 𝗗𝘂𝗮𝗹 𝗚𝗲𝗻𝗲𝘁𝗶𝗰 𝗔𝗽𝗽𝗿𝗼𝗮𝗰𝗵 𝗘𝗻𝗵𝗮𝗻𝗰𝗲𝘀 𝗧𝗿𝗲𝗮𝘁𝗺𝗲𝗻𝘁 𝗼𝗳 𝗦𝗽𝗶𝗻𝗮𝗹 𝗠𝘂𝘀𝗰𝘂𝗹𝗮𝗿 𝗔𝘁𝗿𝗼𝗽𝗵𝘆 * Researchers have developed a novel strategy to treat spinal muscular atrophy (SMA) by combining gene supplementation with CRISPR genome editing. The approach aims to address the limitations of current therapies that only offer temporary relief. The study employs a CRISPR-Cas9-based homology-independent targeted integration (HITI) strategy to correct the SMA mutation in mice in conjunction with Smn1 cDNA supplementation. HITI, effective in both dividing and non-dividing cells, uses non-homologous end joining (NHEJ) to integrate transgenes, ensuring stable gene correction without the need for homologous recombination. The researchers targeted intronic sequences upstream of exon 2 of the Smn1 gene to avoid deleterious insertions and deletions, incorporating a codon-optimised Smn1 cDNA to restore functional SMN protein production. AAV-PHP.eB capsids were utilised for their superior transduction efficiency, delivering the genetic constructs to motor neurons and peripheral tissues. Continue reading how the new treatment helped mice restore motor capabilities at CRISPR Medicine News 👇  https://lnkd.in/dPvv_YwW #crisprmedicinenews #crisprmedicine #crispr #geneediting #genomeediting Juan Carlos Izpisua Belmonte Altos Labs Salk Institute for Biological Studies

🌟Celebrating the 2024 Nobel Prize in Medicine: MicroRNA and Gene Regulation 🌟 Congratulations to Victor Ambros and Gary Ruvkun, recipients of the 2024 Nobel Prize in Physiology or Medicine, for their pioneering research on microRNA (miRNA). Their discovery, which began with studies on the tiny C. elegans worm, has transformed our understanding of gene regulation. MicroRNAs are small RNA molecules that don’t code for proteins but instead regulate gene expression by binding to messenger RNA (mRNA). This interaction either blocks the mRNA from translating into proteins or leads to its degradation. Their ability to target multiple genes makes miRNAs essential players in complex gene networks, involved in everything from cell development to disease resistance.What makes miRNA particularly significant among other gene regulators, like siRNA and regulatory genes? Unlike other molecules, miRNAs control genes after transcription providing an extra layer of fine-tuned regulation across species. Today, hundreds of different microRNAs in humans influence over half of our protein-coding genes. This discovery is opening doors to potential new treatments for diseases where abnormal miRNA activity is involved, such as cancer.Ambros and Ruvkun’s work reminds us of the power of curiosity-driven research. What started as an investigation into the genes of a small worm has led to profound implications for medicine and the potential for innovative therapeutic approaches. #NobelPrize2024 #Ruvkun #Ambros #GeneRegulation #microRNA #Medicine #Biotechnology #ResearchInnovation #LifeSciences

🔍 Breakthrough in Atherosclerosis Research! 🩺 Recent studies reveal the PTAFR gene as a pivotal player in the development of atherosclerosis (AS), a leading cause of cardiovascular diseases. Through advanced bioinformatics and machine learning techniques, scientists have identified PTAFR as a key regulator influencing neutrophil extracellular traps (NETs), which are crucial in AS pathology. ✨ Key Findings: - Elevated PTAFR levels linked to AS severity. - Potential for PTAFR as a prognostic biomarker for ischemic events. - Inhibition of PTAFR may reduce NET formation, offering new therapeutic avenues. This research highlights the intricate relationship between genetic factors and immune responses, paving the way for innovative treatments that could transform patient outcomes. 👉 Click to learn more about this exciting development! #Atherosclerosis #Biomarkers #CardiovascularHealth #General #InnovationInHealthcare #MedicalResearch #PTAFR #MarketAccess #MarketAccessToday

🌟 2024 Nobel Prize in Medicine: A Breakthrough in Gene Regulation 🧬 The 2024 Nobel Prize in Medicine was awarded to Victor Ambros and Gary Ruvkun for their groundbreaking discovery of microRNAs, a discovery that has revolutionized biotechnology and gene therapy. This breakthrough opens new doors to targeted treatments and therapeutic innovations! 🌱💡 🔬 What are microRNAs (miRNAs)? MicroRNAs are tiny, non-coding RNA molecules that regulate gene expression by binding to messenger RNAs (mRNAs) and controlling their ability to produce proteins. Think of them as fine-tuners of gene activity! 🌱 Why It Matters to Biotechnology: Cancer Treatment: MicroRNA-based therapies show promise in treating cancers by regulating the expression of genes that drive tumor growth. RNA Therapeutics: miRNAs and related RNA technologies, like siRNA (small interfering RNA), are becoming key tools in developing innovative therapies for a range of diseases. Personalized Medicine: With miRNA, we’re one step closer to precision medicine—treating diseases based on individual genetic profiles. 💡 Real-World Impact: This discovery marks a major leap in biotechnology: Gene Regulation: Better understanding of how genes are switched on and off opens possibilities for tackling diseases at the genetic level. Therapeutic Developments: miRNA-based treatments are already paving the way for RNA-based drug therapies, especially for cancers, autoimmune diseases, and genetic disorders. 🔍 Looking Ahead: As biotechnology continues to evolve, the potential of RNA-based therapies is boundless. This Nobel Prize reinforces how crucial fundamental discoveries are in shaping the future of healthcare innovations. 🔬 The future of biotechnology is looking bright with the promise of microRNA-based therapies—more targeted, efficient, and personalized treatments! 🌟 #Biotechnology #NobelPrize #GeneRegulation #MicroRNA #RNAtherapy #InnovationInBiotech #BiotechFuture #MedicalBreakthroughs #biopractify

* 𝗘𝗻𝗵𝗮𝗻𝗰𝗲𝗱 𝗖𝘆𝘁𝗼𝘀𝗶𝗻𝗲 𝗕𝗮𝘀𝗲 𝗘𝗱𝗶𝘁𝗶𝗻𝗴 𝗳𝗼𝗿 𝗗𝘂𝗰𝗵𝗲𝗻𝗻𝗲 𝗠𝘂𝘀𝗰𝘂𝗹𝗮𝗿 𝗗𝘆𝘀𝘁𝗿𝗼𝗽𝗵𝘆 * A novel engineered TadA ortholog-derived cytosine base editor (aTdCBE) can overcome existing limitations in motif preferences and adenosine activity seen in current cytosine base editors (CBEs). The new base editor enables precise cytosine base editing without generating unwanted off-target effects, improving gene editing’s scope and efficiency in treating genetic disorders. The study addresses the challenge of tRNA-specific adenosine deaminase (TadA) variants showing dual activity on cytosine and adenosine bases. Using TadA ortholog screening and multi-sequence alignment, the researchers developed aTdCBE, which lacks adenosine deaminase activity. Continue reading on the CRISPR Medicine News website to learn how the novel CBE was able to correct exon 55 splicing in a humanised mouse model of Duchenne muscular dystrophy (DMD) 👇 https://lnkd.in/dj72r6VZ #crisprmedicinenews #crisprmedicine #crispr #geneediting #genomeediting #dmd #duchennemusculardystrophy #duchenne #baseediting #baseeditor HuidaGene Therapeutics