Comparative dose effectiveness of intravenous and intrathecal AAV9.CB7.hIDS, RGX-121, in mucopolysaccharidosis type II mice
Smith, Miles C., Belur, Lalitha R., Karlen, Andrea D., Erlanson, Olivia, Furcich, Justin, Lund, Troy C., Seelig, Davis, Kitto, Kelley F., Fairbanks, Carolyn A., Kim, Kwi Hye, Buss, Nick, McIvor, R. Scott
Published in Molecular therapy. Methods & clinical development (14.03.2024)
Published in Molecular therapy. Methods & clinical development (14.03.2024)
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