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    An innovative hematopoietic stem cell gene therapy approach benefits CLN1 disease in the mouse model

    An innovative hematopoietic stem cell gene therapy approach benefits CLN1 disease in the mouse model by Marco Peviani, Sabyasachi Das, Janki Patel, Odella Jno‐Charles, Rajesh Kumar, Ana Zguro, Tyler D Mathews, Paolo Cabras, Rita Milazzo, Eleonora Cavalca, Valentina Poletti, Alessandra Biffi

    Published 2023-03-01
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