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Human central nervous system stem cells (HuCNS-SC) are a purified, expanded, and banked population of human neural stem cells originally developed by StemCells, Inc. These multipotent adult stem cells are capable of differentiating into the three primary cell types of the central nervous system: neurons, astrocytes, and oligodendrocytes. HuCNS-SC was investigated for its potential to treat various neurodegenerative conditions through multiple mechanisms, including cell replacement (e.g., myelination in Pelizaeus-Merzbacher disease), enzyme replacement (e.g., providing PPT1 and TPP1 in Neuronal Ceroid Lipofuscinosis), and neuroprotection (e.g., secreting VEGF and phagocytosing debris in retinal degeneration). The cells are typically delivered via direct surgical transplantation into the brain, spinal cord, or subretinal space. While several Phase I/II trials were completed or terminated following the closure of StemCells, Inc. in 2016, research has continued into improved formulations such as Neubright (NB), a single-cell suspension designed for enhanced cell spread and viability in retinal applications.
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