Cellular and molecular approaches to motor neuron therapy in amyotrophic lateral sclerosis and spinal muscular atrophy |
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Authors: | O'Connor Deirdre M Boulis Nicholas M |
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Affiliation: | Department of Neurosurgery, Emory University, 101 Woodruff Circle, Atlanta, GA 30322, United States. |
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Abstract: | Amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy (SMA) are progressive fatal neurodegenerative diseases. They differ in their disease development but have in common a loss of motor neuron as they progress. Research is ongoing to further understand the origin of these diseases but this common thread of motor neuron loss has provided a target for the development of therapies for both ALS and SMA. It is the linked fields of gene and cell therapy that are providing some of the most interesting therapeutic possibilities. |
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Keywords: | Amyotrophic lateral sclerosis Spinal muscular atrophy Gene therapy Stem cells Clinical trials |
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