{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,12]],"date-time":"2026-09-12T04:42:06Z","timestamp":1789188126271,"version":"build-2803163510"},"reference-count":36,"publisher":"Oxford University Press (OUP)","issue":"9","license":[{"start":{"date-parts":[[2009,7,9]],"date-time":"2009-07-09T00:00:00Z","timestamp":1247097600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/academic.oup.com\/journals\/pages\/open_access\/funder_policies\/chorus\/standard_publication_model"}],"funder":[{"name":"MRC, UK"},{"name":"Human Stem Cell Exploratory Center grant from the National Institute of General Medicine","award":["NIH P20 GM075059"],"award-info":[{"award-number":["NIH P20 GM075059"]}]},{"name":"National Institute of Neurological Diseases & Stroke","award":["5 R0l NS055195-04"],"award-info":[{"award-number":["5 R0l NS055195-04"]}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2009,9,1]]},"abstract":"<jats:title>Abstract<\/jats:title>\n               <jats:p>Although we and others have demonstrated that neural stem cells (NSCs) may impact such neurogenetic conditions as lysosomal storage diseases when transplanted at birth, it has remained unclear whether such interventions can impact well-established mid-stage disease, a situation often encountered clinically. Here we report that when NSCs were injected intracranially into the brain of adult symptomatic Sandhoff (Hexb\u2212\/\u2212) mice, cells migrated far from the injection site and integrated into the host cytoarchitecture, restoring \u03b2-hexosaminidase enzyme activity and promoting neuropathologic and behavioral improvement. Mouse lifespan increased, neurological function improved, and disease progression was slowed. These clinical benefits correlated with neuropathological correction at the cellular and molecular levels, reflecting the multiple potential beneficial actions of stem cells, including enzyme cross-correction, cell replacement, tropic support, and direct anti-inflammatory action. Pathotropism (i.e., migration and homing of NSCs to pathological sites) could be imaged in real time by magnetic resonance imaging. Differentially expressed chemokines might play a role in directing the migration of transplanted stem cells to sites of pathology. Significantly, the therapeutic impact of NSCs implanted in even a single location was surprisingly widespread due to both cell migration and enzyme diffusion. Because many of the beneficial actions of NSCs observed in newborn brains were recapitulated in adult brains to the benefit of Sandhoff recipients, NSC-based interventions may also be useful in symptomatic subjects with established disease.<\/jats:p>\n               <jats:p>Disclosure of potential conflicts of interest is found at the end of this article.<\/jats:p>","DOI":"10.1002\/stem.163","type":"journal-article","created":{"date-parts":[[2009,7,10]],"date-time":"2009-07-10T04:11:16Z","timestamp":1247199076000},"page":"2362-2370","source":"Crossref","is-referenced-by-count":43,"title":["Neural Stem Cell Transplantation Benefits a Monogenic Neurometabolic Disorder During the Symptomatic Phase of Disease"],"prefix":"10.1093","volume":"27","author":[{"given":"Mylvaganam","family":"Jeyakumar","sequence":"first","affiliation":[{"name":"Department of Pharmacology, University of Oxford, Oxford, United 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