Engraftment of genetically engineered amniotic epithelial cells corrects lysosomal storage in multiple areas of the brain in mucopolysaccharidosis type VII mice.

@article{Kosuga2001EngraftmentOG,
  title={Engraftment of genetically engineered amniotic epithelial cells corrects lysosomal storage in multiple areas of the brain in mucopolysaccharidosis type VII mice.},
  author={Motomichi Kosuga and Katsunori Sasaki and Akiko Tanabe and X-k Li and Hiroji Okawa and Ikuko Ogino and Osamu Okuda and Hiroyuki Arai and Norio Sakuragawa and Yuko Kamata and Noriyuki Azuma and Seiichi Suzuki and Masao Yamada and Torayuki Okuyama},
  journal={Molecular therapy : the journal of the American Society of Gene Therapy},
  year={2001},
  volume={3 2},
  pages={139-48}
}
Cell-mediated gene therapy for visceral lesions of lysosomal storage diseases is promising; however, the treatment of central nervous system (CNS) lesions remains a challenge. In this study, we generated rat amniotic epithelial cells (AEC) that overexpress and secrete human beta-glucuronidase (GUSB) following transduction with an adenoviral vector encoding human GUSB. The AEC were used as donor cells for cell-mediated gene therapy of CNS lesions in mice with mucopolysaccharidosis type VII… CONTINUE READING