Gene Therapy
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Central nervous system
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A spinal motor neuron-specific enhancer for cell-type specific gene expression control in the developing and adult central nervous system. This technology is gene expression regulatory element that specifically targets cholinergic spinal motor neurons in the central nervous system and can be used for gene expression control throughout the mouse or human lifespan. This technology is a gene enhancer, capable of facilitating either overexpression or knockdown of genes in a highly-specific and temporally-controlled manner in cholinergic motor neurons of the central nervous system.