Engineered nucleic acids and methods of use thereof
First Claim
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1. A pharmaceutical formulation comprising:
- i) an effective amount of a messenger ribonucleic acid (mRNA) sequence encoding a melanocyte-stimulating hormone polypeptide, wherein the mRNA sequence is at least 90% identical to the sequence of SEQ ID NO;
19, and wherein the mRNA sequence comprises an open reading frame encoding SEQ ID NO;
10; and
ii) a pharmaceutically acceptable carrier,wherein the formulation is suitable for repeated administration to a mammalian subject in need thereof.
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Abstract
Provided are compositions and methods for delivering biological moieties such as modified nucleic acids into cells to modulate protein expression. Such compositions and methods include the use of modified messenger RNAs, and are useful to treat or prevent diseases, disorders or conditions, or to improve a subject'"'"'s heath or wellbeing.
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Citations
13 Claims
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1. A pharmaceutical formulation comprising:
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i) an effective amount of a messenger ribonucleic acid (mRNA) sequence encoding a melanocyte-stimulating hormone polypeptide, wherein the mRNA sequence is at least 90% identical to the sequence of SEQ ID NO;
19, and wherein the mRNA sequence comprises an open reading frame encoding SEQ ID NO;
10; andii) a pharmaceutically acceptable carrier, wherein the formulation is suitable for repeated administration to a mammalian subject in need thereof. - View Dependent Claims (2, 3, 4, 5)
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6. A pharmaceutical formulation comprising:
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i) an effective amount of an mRNA sequence encoding a melanocyte-stimulating hormone polypeptide, wherein the mRNA sequence is at least 90% identical to the sequence of SEQ ID NO;
19, and wherein the mRNA sequence comprises an open reading frame encoding SEQ ID NO;
10;ii) a cell penetration agent; and iii) a pharmaceutically acceptable carrier, wherein the formulation is suitable for repeated administration to a mammalian subject in need thereof. - View Dependent Claims (7, 8, 9, 10)
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11. An mRNA sequence encoding a melanocyte-stimulating hormone polypeptide, wherein the mRNA sequence is at least 90% identical to the sequence of SEQ ID NO:
- 19, and wherein the mRNA sequence comprises an open reading frame encoding SEQ ID NO;
10. - View Dependent Claims (12, 13)
- 19, and wherein the mRNA sequence comprises an open reading frame encoding SEQ ID NO;
Specification