Minicircle DNA-based Gene Therapy Coupled With Immune Modulation Permits Long-term Expression of α-L-Iduronidase in Mice With Mucopolysaccharidosis Type I
Titel:
Minicircle DNA-based Gene Therapy Coupled With Immune Modulation Permits Long-term Expression of α-L-Iduronidase in Mice With Mucopolysaccharidosis Type I
Auteur:
Osborn, Mark J McElmurry, Ron T Lees, Christopher J DeFeo, Anthony P Chen, Zhi-Ying Kay, Mark A Naldini, Luigi Freeman, Gordon Tolar, Jakub Blazar, Bruce R