Circular rna vectors comprising modified nucleotides
AssigneeUniversity of Massachusetts
InventorsLi LI, Alejandro FELIX MEJIA
Life of this patent
Estimating the term…
Abstract
Provided is an RNA vector comprising: a permuted group 1 intron/exon comprising a 5′ exon, a 3′ exon, and an intron, the intron comprising a 5′ intron end and a 3′ intron end; and at least one modified nucleotide; wherein the 3′ exon is upstream of the 5′ exon; wherein a portion of the intron including the 3′ intron end is upstream of the 3′ exon; wherein a portion of the intron including the 5′ intron end is downstream of the 5′ exon; and wherein the permuted group 1 intron/exon is active in the presence of the modified nucleotide. Also provided is a DNA vector and methods for expressing a target protein from a circular RNA vector.
Claims17
An RNA vector comprising:
- a permuted group 1 intron/exon comprising a 5′ exon, a 3′ exon, and an intron, the intron comprising a 5′ intron end and a 3′ intron end;
- at least one modified nucleotide selected from pseudouridine or N1-methylpseudouridine;
- and
- a nucleic acid sequence encoding a target protein;
- wherein the 3′ exon is upstream of the 5′ exon;
- wherein a portion of the intron including the 3′ intron end is upstream of the 3′ exon;
- wherein a portion of the intron including the 5′ intron end is downstream of the 5′ exon;
- and
- wherein the permuted group 1 intron/exon is active in the presence of the modified nucleotide.
2-3. (canceled)
5-6. (canceled)
(canceled)
(canceled)
12-13. (canceled)
A composition comprising a circular RNA comprising an internal ribosome entry site (IRES) and a nucleic acid sequence encoding a therapeutic protein greater than 200 amino acids in length, further comprising at least one modified nucleotide selected from pseudouridine or N1-methylpseudouridine vector.
(canceled)
A DNA vector comprising:
- a permuted group 1 intron/exon comprising a 5′ exon, a 3′ exon, and an intron, the intron comprising a 5′ intron end and a 3′ intron end;
- and
- a multiple cloning site;
- wherein the 3′ exon is upstream of the 5′ exon;
- wherein a portion of the intron including the 3′ intron end is upstream of the 3′ exon;
- and
- wherein a portion of the intron including the 5′ intron end is downstream of the 5′ exon.
20-34. (canceled)
Dependencies are inferred from the claim text.