Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells

• Published in: The Journal of clinical investigation Vol. 113; no. 8; pp. 1118 - 1129
• Main Authors: Biffi, Alessandra, De Palma, Michele, Quattrini, Angelo, Del Carro, Ubaldo, Amadio, Stefano, Visigalli, Ilaria, Sessa, Maria, Fasano, Stefania, Brambilla, Riccardo, Marchesini, Sergio, Bordignon, Claudio, Naldini, Luigi
• Format: Journal Article
• Language: English
• Published: United States American Society for Clinical Investigation 01.04.2004
• Subjects: Animals; Biomedical research; Bone marrow; Cell Differentiation; Cell Movement; Disease; Disease Models, Animal; Enzymes; Genes; Genetic Therapy; Hematopoietic Stem Cell Transplantation; Kinases; Lentivirus - genetics; Leukodystrophy, Metachromatic - therapy; Metabolism; Mice; Mice, Inbred C57BL; Motor Activity; Nervous system; Nervous System - cytology; Physiology; Proteins; Stem cell transplantation

Gene-based delivery can establish a sustained supply of therapeutic proteins within the nervous system. For diseases characterized by extensive CNS and peripheral nervous system (PNS) involvement, widespread distribution of the exogenous gene may be required, a challenge to in vivo gene transfer str...