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Bone marrow transplantation has shown promise when administered to late-onset GLD patients. However, the side effects (e.g., graft vs. host disease), harsh conditioning regimens (e.g., myelosuppression), and variable therapeutic effects make this an unsuitable option for infantile GLD patients. We previously reported modest improvements in the twitcher mouse model of GLD after intracerebroventricular (ICV) injections of a low-dose of multipotent stromal cells (MSCs). Goals of this study were to improve bone marrow-derived MSC (BMSC) therapy for GLD by increasing the cell dosage and comparing cell type (e.g., transduced vs. native), treatment timing (e.g., single vs. weekly), and administration route (e.g., ICV vs. intraperitoneal [IP]). Neonatal twitcher mice received (a) 2 \u00d7 105 BMSCs by ICV injection, (b) 1 \u00d7 106 BMSCs by IP injection, (c) weekly IP injections of 1 \u00d7 106 BMSCs, or (d) 1 \u00d7 106 lentiviral-transduced BMSCs overexpressing GALC (GALC-BMSC) by IP injection. All treated mice lived longer than untreated mice. However, the mice receiving peripheral MSC therapy had improved motor function (e.g., hind limb strength and rearing ability), twitching symptoms, and weight compared to both the untreated and ICV-treated mice. Inflammatory cell, globoid cell, and apoptotic cell levels in the sciatic nerves were significantly decreased as a result of the GALC-BMSC or weekly IP injections. The results of this study indicate a promising future for peripheral MSC therapy as a noninvasive, adjunct therapy for patients affected with GLD.<\/jats:p>","DOI":"10.1002\/stem.1397","type":"journal-article","created":{"date-parts":[[2013,4,19]],"date-time":"2013-04-19T07:27:00Z","timestamp":1366356420000},"page":"1523-1534","source":"Crossref","is-referenced-by-count":23,"title":["Multipotent Stromal Cells Alleviate Inflammation, Neuropathology, and Symptoms Associated with Globoid Cell Leukodystrophy in the Twitcher Mouse"],"prefix":"10.1093","volume":"31","author":[{"given":"Brittni A.","family":"Scruggs","sequence":"first","affiliation":[{"name":"Center for Stem Cell Research and Regenerative Medicine, SL-99, New Orleans, Louisiana, USA"},{"name":"Department of Pharmacology, SL-83, New Orleans, Louisiana, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Xiujuan","family":"Zhang","sequence":"additional","affiliation":[{"name":"Center for Stem Cell Research and Regenerative Medicine, SL-99, New Orleans, Louisiana, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Annie C.","family":"Bowles","sequence":"additional","affiliation":[{"name":"Center for Stem Cell Research and Regenerative Medicine, SL-99, New Orleans, Louisiana, USA"},{"name":"Department of Cell and Molecular Biology, Tulane University School of Science and Engineering, New Orleans, Louisiana, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Peter A.","family":"Gold","sequence":"additional","affiliation":[{"name":"Center for Stem Cell Research and Regenerative Medicine, SL-99, New Orleans, Louisiana, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Julie A.","family":"Semon","sequence":"additional","affiliation":[{"name":"Center for Stem Cell Research and Regenerative Medicine, SL-99, New Orleans, Louisiana, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jeanne M.","family":"Fisher-Perkins","sequence":"additional","affiliation":[{"name":"Division of Regenerative Medicine, Tulane University National Primate Center, Covington, Louisiana, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Shijia","family":"Zhang","sequence":"additional","affiliation":[{"name":"Center for Stem Cell Research and Regenerative Medicine, SL-99, New Orleans, Louisiana, USA"},{"name":"Department of Pharmacology, SL-83, New Orleans, Louisiana, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ryan W.","family":"Bonvillain","sequence":"additional","affiliation":[{"name":"Center for Stem Cell Research and Regenerative Medicine, SL-99, New Orleans, Louisiana, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Leann","family":"Myers","sequence":"additional","affiliation":[{"name":"Department of Biostatistics & Bioinformatics, Tulane University School of Public Health and Tropical Medicine, New Orleans, Louisiana, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Su Chen","family":"Li","sequence":"additional","affiliation":[{"name":"Department of Biochemistry and Molecular Biology, SL-43, Tulane University School of Medicine, New Orleans, Louisiana, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Allan V.","family":"Kalueff","sequence":"additional","affiliation":[{"name":"Department of Pharmacology, SL-83, New Orleans, Louisiana, USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Bruce A.","family":"Bunnell","sequence":"additional","affiliation":[{"name":"Center for Stem Cell Research and Regenerative Medicine, SL-99, New Orleans, Louisiana, USA"},{"name":"Department of Pharmacology, SL-83, New Orleans, Louisiana, USA"},{"name":"Division of Regenerative Medicine, Tulane University National Primate Center, Covington, Louisiana, 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