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

por Peviani, Marco, Das, Sabyasachi, Patel, Janki, Jno‐Charles, Odella, Kumar, Rajesh, Zguro, Ana, Mathews, Tyler D, Cabras, Paolo, Milazzo, Rita, Cavalca, Eleonora, Poletti, Valentina, Biffi, Alessandra
Publicado 2023