Microrna-dependent mrna switches for tissue-specific mrna-based therapies
AssigneeCornell University
InventorSeyed Hani Najafi-Shoushtari
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Abstract
Synthetic 5′UTR sequences comprising miRNA binding sites that allow for enhanced translation efficiencies of linked open reading frames, vectors having the synthetic sequences linked to an open reading frame and methods of using isolated mRNA and vectors with the synthetic sequences linked to an open reading frame, are provided.
Claims29
Isolated mRNA comprising a synthetic 5′UTR, a coding sequence for a therapeutic or prophylactic gene product, and a poly A sequence, wherein the synthetic 5′UTR comprises a nucleotide sequence that forms at least a first stem loop or hairpin structure, wherein the at least first stem or portion of the hairpin comprises a first microRNA (miRNA) binding site or a portion thereof, the presence of which in the mRNA results in enhanced translation of the gene product relative to corresponding mRNA that lacks the nucleotide sequence.
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Dependencies are inferred from the claim text.