AGTC Genomics’ Post

microRNAs are a post-transcriptional regulators of gene expression, which are implicated in various biological processes and also can act as biomarkers for pathologicalconditions. In diabetic retinopathy, a body of research demonstrates the role of deregulated miRNAs in the disease pathogenesis by impacting angiognesis as a strictly regulated process abnormally activated in the disease. This research reports the significance of MIR200B polymorphisms in association with sight-thretening diabetic retinopathy. https://lnkd.in/dSmZhcae

Yesterday, EUPATI ensured the coordination of the inaugural meeting of the Multistakeholder Advisory Committee (MAC) for the GEREMY (EU project). 💡 This entity will: ✅ Offer advice/constructive feedback and provide the patient perspective on all aspects of the project, such as the social embedding elements (WP5) as well as the review of guidelines and outputs of the project (WP6 - Preparation for clinical trials and other regulatory aspects) ✅ Raise Consortium Members’ awareness about meaningful patient engagement and its implementing measures on a regular basis This is an innovative approach for patient’s involvement within a very promising project! Since 2023 EUPATI is actively involved in the EU Project “GEREMY”. The Consortium is led by the Netherlands Heart Institute. The project aims to develop a novel approach which aims to deliver the first cardiac Gene Therapy for treatment of rare inherited forms of Arrhythmogenic cardiomyopathy.   EUPATI is supporting the Consortium on different levels, in particular: ✅ Ensure a patient-centred approach ✅Train and guide Consortium members about patient engagement in clinical development ✅Contribute to communication and dissemination activities #GEREMYProject #EUPATI #Collaboration #PatientEngagement

Day 3 of Team Interius making us proud at American Society of Gene & Cell Therapy in Baltimore! Stop by & see some of our poster presentations in person, or view them online: https://lnkd.in/eKiSKA4 Abstract Number 2. In Vivo Generation of Both CAR T Cells and CAR NK Cells Using a CD7 Abstract Number 201. Rational Design of a Detargeted Vesiculovirus Fusogen to Enable Targeted In Vivo Gene Delivery Abstract Number 235. Pharmacokinetics and Vector Shedding in NHPs Following a Single Intravenous Infusion of a CD20-Targeted Engineered Lentiviral Vector Abstract Number 1360. Automating a Lentivirus Infectious Titer Assay (ITA) Abstract Number1361. Evaluation of Residual Host Cell DNA Clearance and Sizing During Production of a Lentiviral Vector Abstract Number 1839. In Vivo Delivery of an Engineered Lentiviral CAR19 Vector for the Treatment of Autoimmune Diseases #asgct #asgct2024 #phillybio

Latest issue!🌸 Identification and characterization of a new potent inhibitor targeting CtBP1/BARS in #melanoma cells ----------------------------- The C-terminal-binding protein 1/brefeldin A ADP-ribosylation substrate (CtBP1/BARS) acts both as an oncogenic transcriptional co-repressor and as a fission inducing protein required for membrane trafficking and Golgi complex partitioning during mitosis, hence for mitotic entry. CtBP1/BARS overexpression, in multiple cancers, has pro-tumorigenic functions regulating gene networks associated with “cancer hallmarks” and malignant behavior. Here, Carmen Valente, Daniela Corda et al. proposed to target the CtBP1/BARS Rossmann fold with small molecules as selective inhibitors of mitotic entry and pro-tumoral transcriptional activities. Full text👇 https://lnkd.in/d3g_PrrX #RossmannFold #Benzenesulfonamide IFO - Istituto Nazionale Tumori Regina Elena - Istituto Dermatologico San Gallicano

Two instead of three! Adeno-associated viral vectors (AAV) are widely used in various gene therapy approaches due to their ability to efficiently deliver genetic material into target cells. The production typically involves HEK cells requiring transfection with (1) the transgene, (2) AAV packaging genes and (3) AV helper genes, split on three separate plasmids. With only two components required, the 2-plasmid-based transfection system presents a simple and cost-effective alternative strategy for successful AAV production (doi:10.1016/S1525-0016(03)00095-9): ✅ helper and packaging genes combined on one plasmid ✅ reduced cost of goods and optimization work ✅ increased efficiency and robustness  ✅ several serotypes available off-the-shelf PlasmidFactory holds the exclusive licence from the German Cancer Research Centre (DKFZ) for global production and distribution of Helper & Packaging plasmids of the pDG and pDP family.   Explore our AAV plasmid portfolio (https://lnkd.in/eynV7eUN)   #2-plasmid-transfection-system #plasmidDNA #AAV #GeneTherapy #PlasmidFactory

Today, we’ll likely see Pfizer’s Beqvez (or another commercial name) get approved for Hemophilia B. + Recall, Hemgenix from CSL is already approved in Hemophilia B as of November 2022. + The addition of Pfizer’s gene therapy will make for the third rare disease category with more than one gene therapy available – the others being Sickle Cell Disease (Lyfgenia and Casgevy) and Transfusion Dependent Beta Thalassemia (Zynteglo and Casgevy). + Competition within the same indication already exists in cell therapy in oncology. + Unlike both of the above-named categories where a CRISPR-based therapy is competing with an AAV-based therapy, both Heme B products will be AAV-based (virally delivered). More to come from Pfizer.... #zolgensma #cgt #genetherapy #hemophilia #hemgenix #csl

We're thrilled to announce that Dr. Byron Lam one of our investigators from our breakthrough clinical trial for Retinitis Pigmentosa patients will be presenting Endogena Therapeutics, Inc.’s latest clinical data at the Ninth Annual Retinal Cell and Gene Therapy Innovation Summit, jointly organized by the Foundation Fighting Blindness and the Oregon Health & Science University and OHSU Casey Eye Institute. 🔬 Presentation Details: 📅 Date: Friday, May 3 🕒 Time: 3:40pm – 3:55pm 📍 Location: Hyatt Regency Seattle, USA 👨⚕️ Presenter: Dr. Byron Lam Dr. Lam will be sharing insights from our first-in-human study evaluating the safety, tolerability, and exploratory efficacy of our photoreceptor regeneration treatment with EA-2353 in patients with retinitis pigmentosa. This is a pivotal moment for Endogena as we continue our mission to revolutionize the treatment of retinal diseases. Dr. Lam's presentation promises to offer invaluable insights for researchers and the science community into our cutting-edge research and the potential impact on patients' lives. Join us in celebrating this milestone and witnessing the future of retinal therapy unfold. Don't miss Dr. Lam's presentation at the summit - it's sure to be a highlight of the event! For more information about the summit and to register, visit: @retina Innovation Summit 2024 #Endogena #RetinalTherapy #InnovationSummit #ClinicalData #RetinitisPigmentosa #MedicalResearch #HealthcareInnovation

14/16 Speaker of #Pezcoller24 Symposium 𝐶𝑎𝑛𝑐𝑒𝑟 𝑎𝑠 𝑎 𝑠𝑦𝑠𝑡𝑒𝑚𝑖𝑐 𝑑𝑖𝑠𝑒𝑎𝑠𝑒: 𝑖𝑛𝑡𝑒𝑟𝑎𝑐𝑡𝑖𝑜𝑛𝑠 𝑏𝑒𝑡𝑤𝑒𝑒𝑛 𝑡𝑢𝑚𝑜𝑟 𝑎𝑛𝑑 ℎ𝑜𝑠𝑡 Trento, June 24-25, 2024 Immune checkpoint blockade (ICB) has shown remarkable efficacy in a subset of patients, but many #cancers remain durably #resistant to ICB. Sophia Guldberg’s results (Matt Spitzer Lab, University of California, San Francisco) identify a novel macrophage population, aTAMs, macrophages expressing Autoimmune Regulator (Aire) gene, and demonstrate that Aire expression in this phenotypically and functionally distinct population plays a critical role in inhibiting anti-tumor T cell responses. 10 days to go! 👇 https://lnkd.in/dfNaYpxT

#Familyhistory of ovarian cancer put you at higher risk. You may want to talk with your healthcare provider about genetic testing. Gene testing can tell you if you have an inherited gene mutation that raises your risk. Consult Our Medical Oncologist. Dr. Sravan Kumar Dubasi Medical and hemato Oncologist Book appointment: 90528 48508 #Drsravankumardubasi #medicalandhematooncologist #oncologist #oralcancer #redpatches #mouthsore #hematooncologist #Services #stomachcancer #lungcancer #livercancer #cervicalcancer #breastcancer #gallbladdercancer #pancreaticcancer #prostatecancer #uterinecancer. Follow us on: https://lnkd.in/gAi2Dkh8

New Series on CRISPR Gene Editing - Part 5 - Applications of CRISPR (Part 2) #MythicalMicrobe #ScienceIlluminated #genetics #genomics #geneediting #crispr #cas9 #crisprcas9 #genes #DNA #mutations #cancer #cancertherapy #cancertreatment #cancerimmunotherapy #immunotherapy #oncogenes #genetherapy #inheriteddiseases #infectiousdiseases #bacteria #antibioticresistance #regenerativemedicine #stemcells #sciencecommunication #sciencefacts #science #research #medicine