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US 10,815,463 B2Granted
Messenger UNA molecules and uses thereof
AssigneeArcturus Therapeutics, Inc.
InventorsPadmanabh Chivukula, Luigi Warren, Kiyoshi Tachikawa, Joseph E. Payne
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Abstract
This invention provides a range of translatable messenger UNA (mUNA) molecules. The mUNA molecules can be translated in vitro and in vivo to provide an active polypeptide or protein, or to provide an immunization agent or vaccine component. The mUNA molecules can be used as an active agent to express an active polypeptide or protein in cells or subjects. Among other things, the mUNA molecules are useful in methods for treating rare diseases.
Claims19
A messenger unlocked nucleic acid (mUNA) molecule, comprising:
- one or more unlocked nucleic acid (UNA) monomers, each independently having a structure of the formula:
- wherein R1 and R2 are each independently H or a phosphodiester linkage,
- Base is a nucleobase selected from the group consisting of uracil, thymine, cytosine, 5-methylcytosine, adenine, guanine, inosine, pseudouracil, 1-methylpseudouracil, and 5-methoxyuracil, and
- R3 is selected from the group consisting of —OR4, SR4, —N(R4)2, —NH(C═O)R4, morpholino, piperazin-1-yl, or 4-alkanoyl-piperazin-1-yl;
- wherein each R4 is independently selected from the group consisting of H, alkyl, a cholesterol, a lipid molecule, a polyamine, an amino acid, and a polypeptide;
- and
- nucleic acid monomers wherein the mUNA molecule is translatable to express a polypeptide or protein.
4 more elements
Dependencies are inferred from the claim text.