Comparative dose effectiveness of intravenous and intrathecal AAV9.CB7.hIDS, RGX-121, in mucopolysaccharidosis type II mice
Titel:
Comparative dose effectiveness of intravenous and intrathecal AAV9.CB7.hIDS, RGX-121, in mucopolysaccharidosis type II mice
Auteur:
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