Motor neuron-specific expression vectors
AssigneeThe Salk Institute for Biological Studies
InventorNeal Dilip Amin
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Abstract
The present disclosure relates to nucleic acid promoter sequences that are able to specifically express genes operatively linked to the promoter in brainstem and spinal motor neuron cells, and to methods for using such promoters to selectively express genes in motor neurons in vitro and in vivo. It is based, at least in part, on the discovery that the nucleic acid of SEQ ID NO: 1 functioned as a motor neuron-specific promoter and was successful in expressing transgenes in motor neuron cells in vivo. The present disclosure also relates to compositions that can increase the activity or expression level of miR-218 and to compositions that can decrease the expression of miR-218 target nucleic acids.
Claims7
A method of treating a human subject diagnosed with or at risk of having a motor neuron disease, comprising administering to a subject in need thereof, a therapeutically effective amount of a composition comprising a nucleic acid encoding a compound that increases miR-218 expression, wherein the nucleic acid encoding a compound that increases miR-218 expression encodes a transcription factor selected from the group consisting of Isl1, Isl2, Lux, Isl-Lhx3 fusion proteins, Phox2a, and a combination thereof.
Dependencies are inferred from the claim text.