Raffaele Fronza’s Post

FutureNeuro Principal Investigator Professor Matt Campbell , wins a €150K ERC Proof of Concept award to develop a novel gene therapy for AMD, a leading cause of blindness. https://lnkd.in/dDebr7x3

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

Stop by Matteo's poster to learn more about the progresses on our mutation-independent adRP program, or the latest build of our editors portfolio.

For patients with Factor VII deficiency, approximately 78% of the disease-associated mutations are missense mutations, with no treatment available to directly address the underlying genetic defect. In this study, Maria Eugenia Chollet presents the result of using stem cell and organoids technology together with gene editing, to correct a common F VII missense mutation and evaluate the recovery of FVII protein production and function in patient-derived cells. #EHA24 #Hematology #FVIIDeficiency

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

For patients with Factor VII deficiency, approximately 78% of the disease-associated mutations are missense mutations, with no treatment available to directly address the underlying genetic defect. In this study, Maria Eugenia Chollet Dugarte presents the result of using stem cell and organoids technology together with gene editing, to correct a common F VII missense mutation and evaluate the recovery of FVII protein production and function in patient-derived cells. #EHA24 #Hematology #FVIIDeficiency

For patients with Factor VII deficiency, approximately 78% of the disease-associated mutations are missense mutations, with no treatment available to directly address the underlying genetic defect. In this study, Maria Eugenia Chollet presents the result of using stem cell and organoids technology together with gene editing, to correct a common F VII missense mutation and evaluate the recovery of FVII protein production and function in patient-derived cells. #EHA24 #Hematology #FVIIDeficiency

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

[RESEARCH] METACHROMATIC LEUKODYSTROPHY: NEW GENE THERAPY PROVES EFFECTIVE IN MICE Metachromatic leukodystrophy is a rare genetic disorder that mainly affects young children and results in severe neurological symptoms accompanied by a loss of motor and intellectual capacities. At Paris Brain Institute, Françoise Piguet and her colleagues have developed a gene therapy treatment capable of correcting the primary anomaly observed in the disease: the accumulation of sulfatides in the brain and spinal cord. Effective in mice, as shown by new results published in Molecular Therapy – Methods & Clinical Development, this technique paves the way for human clinical trials. https://lnkd.in/gP2SBHhy

🔬 𝐄𝐱𝐜𝐢𝐭𝐢𝐧𝐠 𝐑𝐞𝐬𝐞𝐚𝐫𝐜𝐡 𝐀𝐥𝐞𝐫𝐭: 𝐀 𝐃𝐞𝐞𝐩 𝐃𝐢𝐯𝐞 𝐢𝐧𝐭𝐨 𝐀𝐀𝐕5 𝐈𝐧𝐭𝐞𝐠𝐫𝐚𝐭𝐢𝐨𝐧 𝐏𝐫𝐨𝐟𝐢𝐥𝐞𝐬 This study, in collaboration with BioMarin Pharmaceutical Inc., leverages Target Enrichment Sequencing (TES) to explore the safety and integration profiles of AAV5-based gene therapy vectors in mice. 💡 𝐓𝐡𝐞 𝐟𝐢𝐧𝐝𝐢𝐧𝐠𝐬💡 The majority of vector genomes persist in their episomal form. While a minor proportion of the vector genomes do integrate into the host genome at low frequency, primarily within the first week post-treatment, with no evidence of clonal expansion or tumorigenesis over a year-long analysis.  TES was performed by designed RNA bait set (vector-specific baits) to enrich vector-vector as well as vector-genome junctions capturing the entire vector sequence, paving the way for a deep and robust characterization of AAV integration profiles and highlights the method’s capacity to detect clonal outgrowth with high sensitivity. 👉 𝐃𝐢𝐯𝐞 𝐢𝐧𝐭𝐨 𝐭𝐡𝐞 𝐝𝐞𝐭𝐚𝐢𝐥𝐬: https://lnkd.in/egXiXKk9 Would you like to speak to one of our experts? 🤝 Get in touch with Matteo Franco   #WhatWeDoMatters #ProtaGene #GeneTherapy