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) an adjacent exon sequence of a 3′ Group I self-splicing intron-exon, b) an internal ribosome entry site (IRES), c) a protein coding region or noncoding region, and d) an adjacent exon sequence of a 5′ Group I self-splicing intron-exon.
Claims28
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) an internal ribosome entry site (IRES),
- c) a protein coding region or noncoding region, and
- d) an exon sequence of a 5′ Group I self-splicing intron-exon,
- wherein the circular RNA is at least 1,600 nucleotides in size.
A composition comprising:
- a) an effective amount of 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) an internal ribosome entry site (IRES),
- iii) a protein coding region or noncoding region, and
- iv) an exon sequence of a 5′ 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,
- wherein the circular RNA is at least 1,600 nucleotides in size.
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) an internal ribosome entry site (IRES),
- iii) a protein coding region or noncoding region, and
- iv) an exon sequence of a 5′ Group I self-splicing intron-exon;
- and
- b) a pharmaceutically acceptable carrier, excipient or diluent,
- wherein the circular RNA is at least 1,600 nucleotides in size.
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) an internal ribosome entry site (IRES),
- c) a protein coding region or noncoding region, and
- d) an exon sequence of a 5′ Group I self-splicing intron-exon,
- wherein the circular RNA is at least 1,600 nucleotides in size.
Dependencies are inferred from the claim text.