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  1. 1
    The rare disease burden: a multidimensional challenge

    The rare disease burden: a multidimensional challenge by Zuzanna Cyske, Edyta Radzanowska-Alenowicz, Estera Rintz, Lidia Gaffke, Karolina Pierzynowska

    Published 2025-07-01
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  2. 2
    In vivo direct lentiviral gene therapy improves disease pathology in a mucopolysaccharidosis IVA murine model

    In vivo direct lentiviral gene therapy improves disease pathology in a mucopolysaccharidosis IVA murine model by Betul Celik, Estera Rintz, Shaukat Khan, Andrés Felipe Leal, Fnu Nidhi, Shunji Tomatsu

    Published 2025-09-01
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