ArticleStem cells (Dayton, Ohio)2009
Neural stem cell transplantation benefits a monogenic neurometabolic disorder during the symptomatic phase of disease.
Article in Stem cells (Dayton, Ohio), 2009. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 26 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
26 citing papers in PubMed, 45 citations in OpenAlex.
- Article
- Paradigm Shift: Multiple Potential Pathways to Neurodegenerative Dementia.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2023Article
- Brain transplantation of genetically corrected Sanfilippo type B neural stem cells induces partial cross-correction of the disease.Molecular therapy. Methods & clinical development · 2022Article
- Modulation of gene expression on a transcriptome-wide level following human neural stem cell transplantation in aged mouse stroke brains.Experimental neurology · 2022Article
- Neural Stem Cells for Early Ischemic Stroke.International journal of molecular sciences · 2021Review
- Human neural stem cells improve early stage stroke outcome in delayed tissue plasminogen activator-treated aged stroke brains.Experimental neurology · 2020Article
- Human iPSC-Based Models for the Development of Therapeutics Targeting Neurodegenerative Lysosomal Storage Diseases.Frontiers in molecular biosciences · 2020Review
- Application of Stem Cells in Stroke: A Multifactorial Approach.Frontiers in neuroscience · 2020Review
- Bi-functional IgG-lysosomal enzyme fusion proteins for brain drug delivery.Scientific reports · 2019Article
- Neural stem cell therapy for subacute and chronic ischemic stroke.Stem cell research & therapy · 2018Review
- Emptying the stores: lysosomal diseases and therapeutic strategies.Nature reviews. Drug discovery · 2018Review
- The state of the art in stem cell biology and regenerative medicine: the end of the beginning.Pediatric research · 2018Review
- Generation of Human Induced Pluripotent Stem Cell-Derived Bona Fide Neural Stem Cells for Ex Vivo Gene Therapy of Metachromatic Leukodystrophy.Stem cells translational medicine · 2017Article
- Stem Cell Therapy for the Central Nervous System in Lysosomal Storage Diseases.Human gene therapy · 2016Review
- Glial progenitor cell-based treatment of the childhood leukodystrophies.Experimental neurology · 2016Review
- Moving towards effective therapeutic strategies for Neuronal Ceroid Lipofuscinosis.Orphanet journal of rare diseases · 2016Review
- Animal models of GM2 gangliosidosis: utility and limitations.The application of clinical genetics · 2016Review
- Neural stem cell transplantation promotes behavioral recovery in a photothrombosis stroke model.International journal of clinical and experimental pathology · 2015Article
- Human neural stem cells rapidly ameliorate symptomatic inflammation in early-stage ischemic-reperfusion cerebral injury.Stem cell research & therapy · 2014Article
- Clinical translation of human neural stem cells.Stem cell research & therapy · 2013Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
11 authors at 3 institutions in 2 countries.
Funding
Abstract
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(-/-)) mice, cells migrated far from the injection site and integrated into the host cytoarchitecture, restoring beta-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.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.