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.
Claims17
A recombinant nucleic acid comprising a motor neuron-specific promoter nucleic acid sequence comprising SEQ ID NO:1, operatively linked to a first nucleic acid sequence encoding a compound that increases miR-218 expression, wherein the first nucleic acid sequence encoding the compound that increases miR-218 expression is a transcription factor selected from the group consisting of Isl1, Isl2, Lhx3, Isl-Lhx3 fusion proteins, Phox2a, and a combination thereof.
A pharmaceutical composition comprising:
- a recombinant nucleic acid comprising a motor neuron-specific promoter nucleic acid sequence comprising SEQ ID NO:1, operatively linked to a first nucleic acid encoding a compound that increases miR-218 expression, wherein the first nucleic acid sequence encoding a compound that increases miR-218 expression is a transcription factor selected from the group consisting of Isl1, Isl2, Lhx3, Isl-Lhx3 fusion proteins, Phox2a, and a combination thereof;
- and
- a pharmaceutically acceptable carrier.
Dependencies are inferred from the claim text.