Circular RNA for translation in eukaryotic cells
AssigneeMassachusetts Institute of Technology
InventorsDaniel G. Anderson, Robert Alexander Wesselhoeft, Piotr S. Kowalski
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Abstract
Circular RNA and methods and constructs for engineering circular RNA are disclosed. In some embodiments, the circular RNA includes the following elements arranged in the following sequence: a) a 3′ Group I self-splicing intron fragment, b) an internal ribosome entry site (IRES), c) a protein coding region or noncoding region, and d) a 5′ Group I self-splicing intron fragment.
Claims21
A circular RNA comprising the following elements arranged in the following sequence:
- a) an exon sequence of a 3′ Group I self-splicing intron-exon,
- b) a 5′ internal homology region,
- c) an internal ribosome entry site (IRES),
- d) a protein coding region,
- e) a 3′ internal homology region, and
- f) an exon sequence of a 5′ Group I self-splicing intron-exon,
- wherein the 5′ internal homology region, the 3′ internal homology region, or both are exogenous to the Group I self-splicing intron-exon.
A composition, comprising:
- a) an effective amount of a circular RNA, wherein the circular RNA comprises the following elements arranged in the following sequence:
- i) an exon sequence of a 3′ Group I self-splicing intron-exon,
- ii) a 5′ internal homology region,
- iii) an internal ribosome entry site (IRES),
- iv) a protein coding region,
- v) 3′ internal homology region, and
- vi) an exon sequence of a 5′ Group I self-splicing intron-exon, wherein the 5′ internal homology region, the 3′ internal homology region, or both are exogenous to the Group I self-splicing intron-exon;
- and
- b) a nanocarrier selected from the group consisting of a lipid, a polymer and a lipo-polymeric hybrid.
A method of treating a disease, the method comprising administering an effective amount of a circular RNA to a subject in need thereof, wherein the circular RNA comprises the following elements arranged in the following sequence:
- a) an exon sequence of a 3′ Group I self-splicing intron-exon,
- b) a 5′ internal homology region,
- c) an internal ribosome entry site (IRES),
- d) a protein coding region,
- e) a 3′ internal homology region, and
- f) an exon sequence of a 5′ Group I self-splicing intron-exon,
- wherein the 5′ internal homology region, the 3′ internal homology region, or both are exogenous to the Group I self-splicing intron-exon.
Dependencies are inferred from the claim text.