title: Mesenchymal stem cells as cellular vectors for pediatric neurological disorders.
abstract: Lysosomal storage diseases are a heterogeneous group of hereditary disorders characterized by a deficiency in lysosomal function. Although these disorders differ in their etiology and phenotype those that affect the nervous system generally manifest as a profound deterioration in neurologic function with age. Over the past several decades implementation of various treatment regimens including bone marrow and cord blood cell transplantation, enzyme replacement, and substrate reduction therapy have proved effective for managing some clinical manifestations of these diseases but their ability to ameliorate neurologic complications remains unclear. Consequently, there exists a need to develop alternative therapies that more effectively target the central nervous system. Recently, direct intracranial transplantation of tissue-specific stem and progenitor cells has been explored as a means to reconstitute metabolic deficiencies in the CNS. In this chapter we discuss the merits of bone marrow-derived mesenchymal stem cells (MSCs) for this purpose. Originally identified as progenitors of connective tissue cell lineages, recent findings have revealed several novel aspects of MSC biology that make them attractive as therapeutic agents in the CNS. We relate these advances in MSC biology to their utility as cellular vectors for treating neurologic sequelae associated with pediatric neurologic disorders.
authors.affiliation: Department of Molecular Therapeutics, The Scripps Research Institute, 130 Scripps Way, A213, Jupiter, FL 33458, USA. Electronic address: dphinney@scripps.edu.
authors.forename: Donald G
authors.initials: DG
authors.lastname: Phinney
authors.affiliation: Division of Clinical Laboratory Diagnostics, Biology Department, National Dnepropetrovsk University, Dnepropetrovsk, Ukraine.
authors.forename: Iryna A
authors.initials: IA
authors.lastname: Isakova
mesh_terms: D000818:Animals; D001921:Brain / Q000503:physiopathology; D002648:Child; D006801:Humans; D045164:Mesenchymal Stem Cell Transplantation / Q000379:methods*; D059630:Mesenchymal Stem Cells / Q000502:physiology; D009422:Nervous System Diseases / Q000503:physiopathology / Q000628:therapy*
keywords: Cell therapy; Central nervous system; Lysosomal storage disease; Marrow stromal cell; Mesenchymal stem cell; Migration; Paracrine signaling; Pediatric