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.
Claims20
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.
The composition of claim 11, wherein the viral vector is selected from the group consisting of adenovirus, adeno-associated virus, vaccinia, herpesviruses, baculoviruses, retroviruses, parvovirus, and lentivirus.
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;
- and
- a pharmaceutically acceptable carrier.
Dependencies are inferred from the claim text.