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.
Claims33
A circular RNA comprising 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.
The circular RNA of claim, 1 wherein one or more of the 3′ Group I self-splicing intron fragment and the 5′ Group I self-splicing intron fragment is a post-splicing exon sequence.
The circular RNA of claim, 1 wherein one or more of the 3′ Group I self-splicing intron fragment and the 5′ Group I self-splicing intron fragment comprises an adjacent exon sequence.
A composition comprising:
- a) an effective amount of circular RNA, wherein the circular RNA comprises the following elements arranged in the following sequence:
- i) a 3′ Group I self-splicing intron fragment,
- ii) an internal ribosome entry site (IRES),
- iii) a protein coding region or noncoding region, and
- iv) a 5′ Group I self-splicing intron fragment;
- and
- b) a nanocarrier selected from the group consisting of a lipid, a polymer and a lipo-polymeric hybrid.
A composition comprising:
- a) an effective amount of the circular RNA, wherein the circular RNA comprises the following elements arranged in the following sequence:
- i) a 3′ Group I self-splicing intron fragment,
- ii) an internal ribosome entry site (IRES),
- iii) a protein coding region or noncoding region, and
- iv) a 5′ Group I self-splicing intron fragment;
- and
- b) a pharmaceutically acceptable carrier, excipient or diluent.
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) 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.
Dependencies are inferred from the claim text.