US2023277690A1PendingUtilityA1

Motor neuron-specific expression vectors

Assignee: SALK INST FOR BIOLOGICAL STUDIPriority: May 26, 2015Filed: Apr 7, 2023Published: Sep 7, 2023
Est. expiryMay 26, 2035(~8.8 yrs left)· nominal 20-yr term from priority
Inventors:Neal Dilip Amin
C12N 2310/20A61K 48/0066A01K 67/0275A61K 31/7088C12N 15/85A01K 67/0276C12N 15/113A61K 31/7105A61K 31/713A61P 25/28A61K 48/0016A01K 2217/052A01K 2217/075A01K 2227/105A01K 2267/0318A01K 2267/0393A01K 2227/50C12N 2830/008C12N 2310/113Y02A50/30
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Claims

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.

Claims

exact text as granted — not AI-modified
What is claimed is: 
     
         1 . A recombinant nucleic acid comprising a motor neuron-specific promoter nucleic acid sequence comprising SEQ ID NO:1, operatively linked to a fluorescent protein. 
     
     
         2 . The recombinant nucleic acid of  claim 1 , wherein the recombinant nucleic acid is comprised in a viral vector. 
     
     
         3 . The recombinant nucleic acid of  claim 2 , wherein the viral vector is an adenovirus, adeno-associated virus, vaccinia, herpesviruses, baculoviruses, retroviruses, parvovirus, or lentivirus. 
     
     
         4 . The recombinant nucleic acid of  claim 1 , wherein the fluorescent protein is green fluorescent protein (GFP), blue fluorescent protein (BFP), cyan fluorescent protein (CFP), or yellow fluorescent protein (YFP).

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