Long-Term Safety, Immunologic Response, and Imaging Outcomes following Neural Stem Cell Transplantation for Pelizaeus-Merzbacher Disease

• Published in: Stem cell reports Vol. 13; no. 2; pp. 254 - 261
• Main Authors: Gupta, Nalin, Henry, Roland G., Kang, Sang-Mo, Strober, Jonathan, Lim, Daniel A., Ryan, Tamara, Perry, Rachel, Farrell, Jody, Ulman, Mary, Rajalingam, Raja, Gage, Allyson, Huhn, Stephen L., Barkovich, A. James, Rowitch, David H.
• Format: Journal Article
• Language: English
• Published: United States Elsevier Inc 13.08.2019; Elsevier
• Subjects: Brain - diagnostic imaging; Brain - physiology; Child, Preschool; Follow-Up Studies; HLA Antigens - immunology; Humans; Infant; Isoantibodies - blood; leukodystrophy; Magnetic Resonance Imaging; Male; myelin; neural stem cells; Neural Stem Cells - cytology; Neural Stem Cells - metabolism; Neural Stem Cells - transplantation; neurodegenerative disease; oligodendrocyte; pediatric; Pelizaeus-Merzbacher disease; Pelizaeus-Merzbacher Disease - immunology; Pelizaeus-Merzbacher Disease - pathology; Pelizaeus-Merzbacher Disease - therapy; Severity of Illness Index; Stem Cell Transplantation - adverse effects; transplantation; Transplantation, Homologous; Treatment Outcome; myelin; Pelizaeus-Merzbacher disease; neurodegenerative disease; neural stem cells; oligodendrocyte; transplantation; leukodystrophy; pediatric

Four boys with Pelizaeus-Merzbacher disease, an X-linked leukodystrophy, underwent transplantation with human allogeneic central nervous system stem cells (HuCNS-SC). Subsequently, all subjects were followed for an additional 4 years in this separate follow-up study to evaluate safety, neurologic fu...