Bionano’s Post

🚨📅April 17th, Sonia Albini, PhD, Researcher in the Progressive Muscular Dystrophies team at Genethon is attending to the 4th Gene Therapy for Muscular Disorders Summit, Boston MA, for a seminar (09 :30 AM): Making gene therapies for muscular disorders a reality. Don’t miss her presentation about: “Developing MYOrganoids as a Translational Model to Evaluate Gene Therapy Products for DMD”. Following points will be discussed such as : 👉Highlighting the urgent need to develop in vitro models mimicking human DMD phenotypes 👉Using 3D cultures and biomaterials to allow the reconstitution of tissue architecture and microenvironment essential for pathophysiological studies 👉Using DMD-derived MYOrganoids to evaluate the therapeutic efficiency of micro-dystrophin in proof-of-concept studies. Find out more: https://vu.fr/PtpdK #genethon #rarediseases #GeneTherapy #DMD #duchenne

A new strategy allows for high-accuracy assembly of #CRISPR arrays, facilitating multi-target CRISPR applications. Introduced in a recent research paper, the approach streamlines the assembly process, enabling the efficient integration of multiple CRISPR #RNAs (12 for AsCas12a and 15 for RfxCas13d) in a single reaction. The study highlights the differential expression patterns of CRISPR arrays driven by Pol II versus Pol III promoters, which can be leveraged for targeted gene therapy. The authors emphasize the potential of this method to dissect complex cellular networks and support advancements in multi-target gene therapies. 🌐 Source: Xiangtong Zhao et al. | Molecular Therapy Nucleic Acids 📷 Image credit: Miroslaw Miras | Pixabay

StitchR Tackles Muscular Dystrophy Treating muscular dystrophies has long been hindered by the size of the genes involved—too large for current delivery methods; however, StitchR (short for "Stitch RNA") overcomes this challenge through an innovative gene therapy approach that enables the delivery and functional expression of large genes by leveraging RNA repair pathways. A breakthrough technology that delivers two halves of a gene separately. Inside cells, these fragments seamlessly unite, restoring the production of vital proteins like Dystrophin (absent in Duchenne muscular dystrophy) and Dysferlin (lacking in limb girdle muscular dystrophy). It restores therapeutic protein levels to normal in animal models of muscular dystrophy by utilizing a unique RNA-ligation mechanism that ensures the production of only full-length, functional proteins, distinguishing it from other approaches. #GeneTherapy #Biotechnology #MuscularDystrophy #RNAResearch #InnovationInScience #MedicalBreakthrough #RNARepair #MolecularBiology #Research #RNAInnovation #GeneticEngineering #RareDiseases #MolecularTherapy #FutureOfMedicine #BiotechBreakthrough #GeneEditing #ScientificDiscovery #RNARepair #LifeSciences #MedicalResearch #InnovationInBiotech #TherapeuticAdvances #GenomicsRevolution #DiseaseCure

Excited and honored to receive a Certificate of Recognition from the International Journal of Research and Analytical Reviews (IJRAR) for the publication of my paper entitled 'Use of CRISPR Technologies in Duchenne Muscular Dystrophy Gene Therapy' in Volume 11, Issue 4 (December 2024). Grateful for the opportunity to contribute to this significant field of genetic research and gene therapy. Looking forward to further exploring innovations in healthcare and advancing scientific knowledge. Published in a UGC Approved Journal with an Impact Factor of 7.17. #CRISPR #GeneTherapy #DuchenneMuscularDystrophy #IJRAR #Research #Innovation #SustainableDevelopment"

Exciting progress from a UF Innovate | Accelerate client! Solid Biosciences, which expanded its impact and capabilities by acquiring University of Florida startup AavantiBio, is collaborating with the Mayo Clinic to advance cutting-edge gene therapy solutions for cardiac genetic conditions. 🔬 This partnership focuses on creating next-generation therapies for life-threatening cardiac conditions, combining Mayo Clinic's Suppression-Replacement ("Sup-Rep") platform with Solid’s innovative AAV gene therapy technologies. Together, they are paving the way for breakthroughs in cardiac precision medicine to address high unmet needs and improve patient outcomes. 👨🔬⚕️ https://lnkd.in/eb72_CRi #UFInnovate #Innovation #GatorMade

Come and join Scribe Therapeutics team at @ASGCT Conference: Cécile Fortuny is giving a talk on the allele-specific editing of the P23H rhodopsin gene in P23H.RHO-related autosomal dominant retinitis pigmentosa animal model, Addison Wright will be demonstrating the potent editing activity of Scribe’s CasX (CasXE) in non-human primates, and Jason Fernandes will guide you through a comprehensive engineering approach to creating highly potent CasX-Based Repressor to Create Highly Potent Epigenetic Editors (ELXRs). #FutureOfMedicine #GeneEditing #EpigeneticModulation.

𝐍𝐞𝐰 𝐆𝐞𝐧𝐞 𝐓𝐡𝐞𝐫𝐚𝐩𝐲 𝐂𝐨𝐮𝐥𝐝 𝐇𝐚𝐥𝐭 𝐚𝐧𝐝 𝐑𝐞𝐯𝐞𝐫𝐬𝐞 𝐌𝐮𝐬𝐜𝐮𝐥𝐚𝐫 𝐃𝐲𝐬𝐭𝐫𝐨𝐩𝐡𝐲 A new gene therapy for 𝐃𝐮𝐜𝐡𝐞𝐧𝐧𝐞 𝐦𝐮𝐬𝐜𝐮𝐥𝐚𝐫 𝐝𝐲𝐬𝐭𝐫𝐨𝐩𝐡𝐲 (𝐃𝐌𝐃), developed by researchers at the University of Washington School of Medicine, shows promising results in 𝐡𝐚𝐥𝐭𝐢𝐧𝐠 𝐚𝐧𝐝 𝐩𝐨𝐭𝐞𝐧𝐭𝐢𝐚𝐥𝐥𝐲 𝐫𝐞𝐩𝐚𝐢𝐫𝐢𝐧𝐠 𝐦𝐮𝐬𝐜𝐥𝐞 𝐝𝐞𝐭𝐞𝐫𝐢𝐨𝐫𝐚𝐭𝐢𝐨𝐧 𝐜𝐚𝐮𝐬𝐞𝐝 𝐛𝐲 𝐭𝐡𝐞 𝐝𝐢𝐬𝐞𝐚𝐬𝐞. The innovative treatment involves delivering protein packets via multiple shuttle vectors to replace the defective dystrophin gene in muscle cells. The 𝐌𝐮𝐬𝐜𝐮𝐥𝐚𝐫 𝐃𝐲𝐬𝐭𝐫𝐨𝐩𝐡𝐲 𝐀𝐬𝐬𝐨𝐜𝐢𝐚𝐭𝐢𝐨𝐧 continues to support the research and efforts are ongoing to advance the therapy through clinical development. Check out our latest market report to gain valuable insight into key trends, recent technological breakthroughs in the DMD treatment pipeline, and ongoing clinical trials that improve accessibility and precision. 𝐃𝐮𝐜𝐡𝐞𝐧𝐧𝐞 𝐦𝐮𝐬𝐜𝐮𝐥𝐚𝐫 𝐝𝐲𝐬𝐭𝐫𝐨𝐩𝐡𝐲 (𝐃𝐌𝐃) 𝐌𝐚𝐫𝐤𝐞𝐭 𝐑𝐞𝐩𝐨𝐫𝐭 𝐋𝐢𝐧𝐤: https://lnkd.in/g2St3XYR #GeneTherapy #MuscularDystrophy #MedicalResearch #DMDTreatment #Biotechnology #HealthcareInnovation #UWMedicine #FutureOfMedicine #GeneticResearch #WissenResearch

At Eppendorf, we trust the power of collaborations, especially when it comes to cell and gene therapy research 🧬 . As the production of cell and gene therapies grows, so does the need for controlled and well-defined processes. 📚 In this bioprocessing resource guide, you will find a collection of case studies and interviews demonstrating the huge potential of stirred-tank bioreactor systems for accelerating cell culture development while optimizing the time spent on research. We're all set to help you elevate your bioprocessing operation to the next level 📈 . Always open to listening and learning from your specific challenges. Let’s bioprocess together. Find out more here: https://lnkd.in/gsQVfknN #bioprocess #customercentricity #12daysofeppendorfbioprocess

🎉 After years of dedicated effort, I am proud to announce that our manuscript is finally published: “Comprehensive Analysis Of Off-Target And On-Target Effects Resulting From Liver-Directed CRISPR-Cas9–Mediated Gene Targeting With Adeno-Associated Viral Vectors” in Molecular Therapy: Methods & Clinical Development. This work demonstrates that AAV-mediated CRISPR targeting in neonatal mouse livers can achieve precise gene editing with low off-target risks. Our findings support S-EPTS/LM-PCR as a potential platform for thorough on- and off-target analyses when using AAV vectors. Many thanks to my co-authors, Thierry VandenDriessche and Kshitiz Singh, as well as the team at ProtaGene GmbH, where much of this work began. https://lnkd.in/e6AUyPnm #GeneTherapy #CRISPR #AAV #GeneEditing #MolecularTherapy #PrecisionMedicine #ProtaGene

Alia Therapeutics is present at ESGCT Rome, the 31st Annual Meeting of the European Society of Gene & Cell Therapy. Meet Matteo Bello, PhD candidate at Alia Therapeutics and at Università di Trento, presenting poster on “A mutation-independent approach to treat autosomal dominant retinitis pigmentosa exploiting compact CRISPR effectors from the human microbiome”. Poster session III - Gene editing: Thursday October 24th between 14-15.30, Poster 619 Alia Therapeutics has a vision to revolutionize medicine by curing genetic diseases at their roots. Our thesis: The diversity and complexity of human genetic diseases demands a customized approach and relying on a limited set of enzymes wouldn’t suffice. Alia’s versatile portfolio of novel CRISPR systems expands the range of targetable genomics sites and increases the precision of genetic modifications, bringing gene editing to the next level of specificity and safety, which is required for therapeutic applications. Learn more at https://lnkd.in/dhVhCzw4 #ESGCT #rome #genetherapy #geneediting #CRISPR #retinitispigmentosa