Enhancer Restricts Transgene Expression to Motor Neurons for Improved Motor Neuron-Targeted Gene Therapy
This invention describes a DNA construct for gene expression in spinal motor neurons enabling gene therapy specific for motor neuron diseases.
Identification of a conserved 125 base-pair Hb9 enhancer that specifies gene expression to spinal motor neurons. Dev. Biol. 293(2):474-85. Human embryonic stem cell-derived motor neurons expressing SOD1 mutants exhibit typical signs of motor neuron degeneration linked to ALS. Dis. Model Mech. 2:189-95, 2009.
Telomerase immortalization of neuronally restricted progenitor cells derived from the human fetal spinal cord. Nature Biotech 22(3):297-305.
Enhancer-specified GFP-based FACS purification of human spinal motor neurons from embryonic stem cells. Exp Neurol. 196(2):224-34.
Directed differentiation of human induced pluripotent stem cells (iPS) generates active motor neurons. Stem Cells 27:806-811, 2009.
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