Nanoscope Therapeutics Inc.’s Post

🔹Light responsive retinal organoids for accurate prediction of clinical outcomes Human retinal organoids that are iPSC derived can recapitulate the complex structure of the human retina with laminar cell organisation mimicking embryonic development. They contain the outer photoreceptor segment of the retina that responds to light. These commercially licensed models that mimic the complex structure of the human retina, provides a powerful tool for understanding retinal diseases, drug safety, and gene therapy evaluation. Carefully designed studies using isogenic controls can provide a solid set of data to progress therapies through clinical translation. Learn more about how our cutting-edge retinal organoids can advance vision research 👉 https://lnkd.in/eZ4eraE4 #Newcells #Retina #VisionResearch #BiotechInnovation

With no treatments currently approved for X-linked retinitis pigmentosa (XLRP), those living with this disease face the inevitable deterioration of their sight. The American Journal of Ophthalmology recently published data from our Phase 1/2 study evaluating our gene therapy in patients with #XLRP. Learn how we are working to make a difference for these patients: https://lnkd.in/dDzyTDHE

At QuTEM (formely Vironova BioAnalytics) we offer a multitude of analysis for Lentiviral vectors, including morphological assessment, size, integrity, purity, aggregation, and Custom classification (e.g. maturation).   The integrity of lentiviral vectors and the presence of intact genomic core structures are indicators of the product's potency and efficacy. TEM plays a crucial role in identifying impurities such as cellular debris and proteins, which can be harmful during transduction. Furthermore, TEM facilitates the assessment of product stability by analysing vector size and aggregation. By unravelling the intricate details of lentiviral particles at the nanoscale level, you can gain valuable insights crucial for optimizing vector design and enhancing therapeutic efficacy.  Spearheaded by Luigi Naldini, the pioneering use of lentiviral #vectors has propelled advancements in gene therapy research. Experimental applications have shown promise in treating a range of diseases, from #diabetes mellitus to prostate cancer, showcasing the potential of this innovative approach. https://lnkd.in/dpmEMm7m #TEM #QuTEM #genetherapy #lenti #innovation

Orchard Therapeutics - U.S., 𝐏𝐫𝐞𝐬𝐞𝐧𝐭𝐬 𝐆𝐫𝐨𝐮𝐧𝐝𝐛𝐫𝐞𝐚𝐤𝐢𝐧𝐠 𝐃𝐚𝐭𝐚 𝐨𝐧 𝐇𝐒𝐂 𝐆𝐞𝐧𝐞 𝐓𝐡𝐞𝐫𝐚𝐩𝐲! We’re excited to share multiple data presentations at the ESGCT 31st Annual Congress in Rome, showcasing the transformative potential of 𝐡𝐞𝐦𝐚𝐭𝐨𝐩𝐨𝐢𝐞𝐭𝐢𝐜 𝐬𝐭𝐞𝐦 𝐜𝐞𝐥𝐥 (𝐇𝐒𝐂) 𝐠𝐞𝐧𝐞 𝐭𝐡𝐞𝐫𝐚𝐩𝐲 for rare neurometabolic diseases. Presentations span metachromatic leukodystrophy (MLD), MPS-IH, and MPS-IIIA, plus new insights for autoimmune disorders and Crohn's disease. https://lnkd.in/e78ArqW7 #genetherapy #biotech #healthcareinnovation #neurometabolicdisorders #orchardtherapeutics #kyowakirin #generesearch #medicaladvancements #rome2024 #raredisease #scienceinnovation

Take your cell and gene therapy research to new heights with cutting-edge solutions designed to continuously measure changes in the concentration of glucose and lactate in cells in culture and facilitate quantitative analysis of cells cultured in 2D and 3D environments. Learn more: https://lnkd.in/ga2Ap8Zg #CellandGeneTherapy #CellTherapy #GeneTherapy #Immunotherapy #Oncology

📍 Multilayered metabolic heterogeneity across tumour types    Zhou et al. constructed a metabolic gene expression atlas using scRNA-seq datasets and investigated multilayered metabolic heterogeneity across different cancers.    Key findings  🔹Shared glycolysis upregulation and divergent regulation of citric acid cycle across different cell types  🔹A colorectal cancer-specific MMP associated with resistance to the cuproptosis inducer elesclomol  🔹The stratification of patients into distinct prognostic subtypes based on metabolic properties of specific cell types, such as myeloid cells. ➡️ More details: https://lnkd.in/emU8-2ZX #ChineseAcademyOfMedicalSciences #PekingUnionMedicalCollege #spatialomics #spatialbiology #singlecellanalysis #singlecell

Ophthopedia Update:Phase 1/2 AAV5-hRKp.RPGR (Botaretigene Sparoparvovec) Gene Therapy: Safety and Efficacy in RPGR-associated X-linked Retinitis Pigmentosa: Retinitis pigmentosa (RP) encompasses a group of inherited degenerative diseases of the retina that affect approximately 1 in 3000 to 4000 persons.1,2 Affected individuals initially experience nyctalopia followed by progressive visual field (VF) constriction leading to tunnel vision, legal blindness, and often, complete blindness.3,4 #Ophthalmology #Ophthotwitter #Scicomm

Pleased to share our new findings as a pre-print in the bioRxiv, which focuses on the study of pediatric high-grade gliomas (pHGGs) harboring the H3.3-G34R mutation and remaining incurable, with a 5-year overall survival of less than 20%. We have utilized a genetically engineered immunocompetent mouse model for this subtype of pHGG. the expression of H3.3-G34R modifies the histone marks deposited at the regulatory elements of genes belonging to the JAK/STAT pathway, leading to increased activation of this pathway. This histone G34R-mediated epigenetic modifications lead to changes in the tumor immune microenvironment of these tumors, towards an immune-permissive phenotype, making these gliomas susceptible to TK/Flt3L immune-stimulatory gene therapy. The application of this therapeutic approach increased the median survival of H3.3-G34R tumor-bearing animals while stimulating the development of anti-tumor immune responses and immunological memory. Our data suggests that the proposed immune-mediated gene therapy has the potential for clinical translation for the treatment of patients harboring H3.3-G34R high-grade gliomas. We published this as shared first authors. #pediatriccancer #pediatricresearch #dna #cell #immunity #mutation #brain #genetherapy

Take your cell and gene therapy research to new heights with cutting-edge solutions designed to continuously measure changes in the concentration of glucose and lactate in cells in culture and facilitate quantitative analysis of cells cultured in 2D and 3D environments. Learn more: https://lnkd.in/ga2Ap8Zg #CellandGeneTherapy #CellTherapy #GeneTherapy #Immunotherapy #Oncology

Take your cell and gene therapy research to new heights with cutting-edge solutions designed to continuously measure changes in the concentration of glucose and lactate in cells in culture and facilitate quantitative analysis of cells cultured in 2D and 3D environments. Learn more: https://lnkd.in/ga2Ap8Zg #CellandGeneTherapy #CellTherapy #GeneTherapy #Immunotherapy #Oncology