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- W2416440869 abstract "Amyotrophic lateral sclerosis (ALS) is a devastating neuromuscular disorder with rapid loss of motor function and death within 5 years of diagnosis. The pathophysiology is complex, but various types of stem cells have properties that make them well suited for treating this disease. Proof-of-principle studies in rodent models of ALS have demonstrated improvements in motor function and survival, although we have yet to reach a cure. These studies have formed the basis of 10 published and 21 planned/ongoing clinical trials. These trials are primarily safety studies and have not yielded significant evidence of therapeutic efficacy to date. Importantly, while animal and in vitro studies have suggested that cells from healthy individuals are superior to cells from diseased sources, the majority of clinical trials have used autologous transplantation. This fact may account for some of the lack of therapeutic benefit observed. However, the future of stem cell therapy for ALS may not lie in the simple autologous or allogeneic transplantation of cells. Instead, the field may move toward the use of genetically modified cells, amplifying the power of these cells to treat the disease and perhaps making moot the question of allogeneic versus autologous transplantation entirely." @default.
- W2416440869 created "2016-06-24" @default.
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- W2416440869 date "2016-01-01" @default.
- W2416440869 modified "2023-09-23" @default.
- W2416440869 title "Stem Cells for Amyotrophic Lateral Sclerosis" @default.
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- W2416440869 doi "https://doi.org/10.1007/978-1-4899-7654-3_13" @default.
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