Of the ~300 million people around the world with a genetic disease, an estimated 10% have a nonsense mutation that causes a premature stop codon. Our CEO Michelle Werner shares with Daniel Levine how Alltrna is developing tRNA therapeutics as universal medicines for these diseases and why a single medicine has the potential to work across nonsense mutations regardless of the gene in which they occur. In this #podcast episode of #RARECast from #GlobalGenes, hear more about how Alltrna designs their tRNA medicines and why this therapeutic approach has the potential to address so many diseases at once. Listen here or read the interview transcript: https://lnkd.in/gV-CQJYk #RNA #tRNA #tRNAbiology #rarediseases #geneticmedicines #StopCodonDisease #tRNAmedicines #machinelearning #AI
Alltrna’s Post
More Relevant Posts
-
Exciting developments in the #FSHD treatment pipeline! While most advanced FSHD programs have faced setbacks as they aim to reduce muscle cell death, the next wave of emerging companies are introducing innovative approaches, focusing on targeting the disease's genetic root, the DUX4 gene. At least eight companies are now aligning behind this strategy, employing various therapeutic methods such as antibody-oligonucleotide conjugates and epigenome modifiers. Check out our latest Rare Disease Spotlight to learn about new approaches targeting the genetic root of the disease - free with registration: https://ow.ly/qYpb50QHe7f Follow BioCentury's coverage on Rare Diseases: https://ow.ly/qFMK50QHe7g #MedicalResearch #MedicalBreakthroughs #GeneticResearch #RareDisease #HealthcareInnovation
To view or add a comment, sign in
-
Genetic medicines are ushering in novel ways of addressing certain diseases. By silencing defective genes, adding working genes, or even editing harmful gene variants, they hold the opportunity to develop potential treatments that may address the root cause of disease. Read our article with Forbes to see how these technologies enable us to push the boundaries of science in hematology research and beyond. https://bit.ly/4ddMkuO
To view or add a comment, sign in
-
Exciting genetic discoveries are offering new hope for targeted therapies for lupus. Researchers have pinpointed mutations in the UNC93B1 gene, which plays a crucial role in our immune system. This could lead to innovative treatments that "reset" the immune system, reducing reliance on traditional immunosuppressants and improving patients' quality of life. These advancements bring us closer to more effective and personalized therapies for lupus and other autoimmune diseases. Stay tuned for more updates on this game-changing research! Opal Bio Pharma (OBP) #LupusAwareness #GeneticResearch #HealthcareInnovation #AutoimmuneDisease #MedicalBreakthrough
To view or add a comment, sign in
-
Join us for Part 2 of our 'Gene Dive' series, where we delve deeper into the fascinating world of Hyperparathyroidism. In this segment, we will unravel the significance of the TRPV6 gene and its counterparts. The TRPV6 gene encodes a calcium-selective channel that plays a critical role in calcium transport and absorption. Explore a spectrum of genes related to Hyperparathyroidism: **Gene Mutations:** Mutations affecting the surface expression of TRPV6 are associated with the upregulation of serine proteases in the placenta of an infant. TRPV6 mutations could also trigger non-alcoholic chronic pancreatitis. Contact us to gain a comprehensive understanding of these gene roles and explore Dhiti's extensive endocrine panel. #GeneExploration #Hyperparathyroidism #GeneStudy #EndocrineDisorders #SpotlightDiseaseOfTheWeek #DhitiOmics #TRPV6gene
To view or add a comment, sign in
-
Enhancers play an important role in determining how genes are expressed in different cells in the body. The goal of the ENHPATHY consortium is to identify these enhancers and how they regulate gene expression. Knowledge about enhancer function may eventually lead to new treatments against diseases associated with enhancer dysfunction, as Dr Salvatore Spicuglia explains. https://lnkd.in/eZXHpcaC #Research #Gene #Enhancers #Disease #Treatment
To view or add a comment, sign in
-
Did you know that RNA interference (RNAi) is a naturally occurring cellular process that’s already at work inside your body? It has two primary functions – to regulate gene expression (i.e., making new proteins) and viral defense. Alnylam scientists have harnessed this natural process to treat specific diseases. Are you #ScienceCurious to know more? Read this Nature Portfolio article (sponsored content) to learn how RNAi therapeutics, and Alnylam, are disrupting disease. https://bit.ly/49YgpNq #RNAi #RNAinterference #siRNA #GeneSilencing #RNAiAmScienceCurious
Alnylam's RNAi Therapeutics are Disrupting Disease
To view or add a comment, sign in
-
Each month, we highlight a gene you can find curated on the Mastermind® Genomic Intelligence Platform. For October, we are featuring THBD! This gene encodes thrombomodulin, a crucial protein involved in regulating blood coagulation. Variants in THBD can lead to issues with clot formation and are associated with an increased risk of thrombosis, which has serious clinical implications, including venous thromboembolism and other blood clotting disorders. To support the research community, we have curated 26,000+ articles on THBD, establishing a comprehensive resource on its role in thrombosis and related conditions. This collection includes cutting-edge research, clinical data, and detailed variant insights. This curated content is available to all Mastermind users. Whether you’re a researcher delving into the intricacies of THBD or a clinician looking for the latest developments, our platform gives you access to an extensive collection of variant data and clinical insights. 🧬 Create a free Mastermind account to explore the platform this invaluable resource and gain a deeper understanding of THBD: https://lnkd.in/g7c_Fzs #THBD #Thrombosis #Genetics #Genomics #Genomenon #MastermindGIP #GeneticResearch #ClinicalResearch
To view or add a comment, sign in
-
The FBN1 gene plays a critical role in producing fibrillin-1, an essential protein that provides structure to connective tissues throughout the body. Mutations in the FBN1 gene are linked to conditions such as Marfan syndrome, which can affect the heart, blood vessels, bones, eyes, and lungs. Early detection through genetic testing can help in managing these conditions. If you or a loved one has a family history of connective tissue disorders, consider speaking to your doctor about genetic testing for FBN1 mutations. Login to know more: https://lnkd.in/dSvYq9g9 #Genetics #FBN1Gene #MarfanSyndrome #GeneticTesting #HealthCare #ConnectiveTissueDisorders #GeneticTesting #PersonalizedMedicine #Mapmygenome #RareDiseases #Mapmygenome #Knowyourself #Genes #DNA #Genomics #Mutations #Diagnosis
To view or add a comment, sign in
-
Inherited retinal diseases (also called inherited retinal dystrophies, or IRDs) are a group of rare eye disorders caused by heritable gene variants associated with disease that can result in vision loss or complete blindness. In recognition of World Retina Day, we are sharing awareness about the importance of genetic testing. Obtaining a confirmed genetic diagnosis through genetic testing is the only way to verify the exact gene variants that are the underlying cause of an IRD—which can inform potential treatment options. #WorldRetinaDay #GeneticTesting
To view or add a comment, sign in
7,007 followers