GENERATION OF HYPERSTABLE mRNAs
AssigneeThe Trustees of the University of Pennsylvania
InventorEric J. Russell
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Abstract
Provided herein is a method for enhancing the stability of a mRNA molecule. Specifically, the invention provides methods of increasing stability or augmenting expression of mRNA or its products by inserting a stability inducing motif at the 3′UTR of the molecule.
Claims76
A hyperstable mRNA, comprising a stability-inducing motif at the 3′UTR of the mRNA, said stability inducing motif comprising a site specific deletion and substitution of a predetermined nucleotide sequence at the 3′UTR.
A method of increasing the stability of a mRNA molecule, comprising the step of inserting a stability inducing motif at the 3′UTR of said mRNA molecule, thereby increasing the stability of a mRNA molecule.
A method of increasing the amount of a mRNA molecule in a cell, comprising the step of inserting a stability inducing motif at the 3′UTR of said mRNA molecule, thereby increasing the amount of a mRNA molecule in a cell.
A method of producing an exogenous protein in a eukaryotic cell, comprising the step of inserting a stability inducing motif at the 3′UTR of a mRNA molecule encoding said protein, thereby producing an exogenous protein in a eukaryotic cell.
A method of treating thalassemia in a subject comprising the step of administering to the subject a DNA construct encoding a hyperstabilized beta-globin mRNA, whereby the hyperstabilized beta-globin mRNA comprises a site specific deletion and substitution of a predetermined nucleotide sequence at the 3′UTR.
A method of treating hemoglobinopathy associated with β-globin comprising the step of administering to the subject a DNA construct encoding a hyperstabilized beta-globin mRNA, whereby the hyperstabilized beta-globin mRNA comprises a site specific deletion and substitution of a predetermined nucleotide sequence at the 3′UTR.
A method of increasing translational efficiency of mRNA in a cell, comprising the step of inserting a stability inducing motif at the 3′UTR of said mRNA molecule, wherein said stability inducing motif comprising a site specific deletion and substitution of a predetermined nucleotide sequence at the 3′UTR.
The method of claim 80, whereby the native β-globin DNA sequence comprises an insertion of the sequence set forth in SEQ ID No.6 at position 561 of the native DNA.
Dependencies are inferred from the claim text.