Publications

Title page of a scientific publication titled 'Symmetric Age-association of Retinal Degeneration in Patients with CLN2-Associated Batten Disease', showing author names and affiliations, abstract, and disclaimers.
Scientific article titled 'Characterization of AAV-mediated dorsal root ganglionopathy' from the journal 'Molecular Therapy: Methods & Clinical Development'.
Scientific article titled 'Recreating pathophysiology of CLN2 disease and demonstrating reversion by TPP1 gene therapy in hiPSC-derived retinal organoids and retina-on-chip.' Includes diagrams of healthy versus CLN2-affected neurons, retinal organoids, and retina-on-chip therapy.
Scientific journal article titled 'Gene therapy ameliorates spontaneous seizures associated with cortical neuron loss in a Cln2R207X mouse model', with authors and affiliations, abstract, and introduction sections.
Scientific research paper titled 'OCT Biomarkers in Ocular CLN2 Disease in Patients Treated with Intravenous Enzyme Replacement Therapy' with author details, publication information, and an abstract discussing retinal degeneration, PR parameters, and optical coherence tomography images.
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