Efficient engraftment of genetically modified cells is necessary to ameliorate central nervous system involvement of murine model of mucopolysaccharidosis type II by hematopoietic stem cell targeted gene therapy

Publication date: Available online 24 June 2020Source: Molecular Genetics and MetabolismAuthor(s): Saori Miwa, Ayako M. Watabe, Yohta Shimada, Takashi Higuchi, Hiroshi Kobayashi, Takahiro Fukuda, Fusao Kato, Hiroyuki Ida, Toya Ohashi
Source: Molecular Genetics and Metabolism - Category: Genetics & Stem Cells Source Type: research