최소 단어 이상 선택하여야 합니다.
최대 10 단어까지만 선택 가능합니다.
다음과 같은 기능을 한번의 로그인으로 사용 할 수 있습니다.
NTIS 바로가기다음과 같은 기능을 한번의 로그인으로 사용 할 수 있습니다.
DataON 바로가기다음과 같은 기능을 한번의 로그인으로 사용 할 수 있습니다.
Edison 바로가기다음과 같은 기능을 한번의 로그인으로 사용 할 수 있습니다.
Kafe 바로가기국가/구분 | United States(US) Patent 등록 |
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국제특허분류(IPC7판) |
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출원번호 | US-0878286 (2015-10-08) |
등록번호 | US-9447164 (2016-09-20) |
발명자 / 주소 |
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출원인 / 주소 |
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대리인 / 주소 |
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인용정보 | 피인용 횟수 : 2 인용 특허 : 393 |
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 imp
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.
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 o
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; andii) a pharmaceutically acceptable carrier,wherein the formulation is suitable for repeated administration to a mammalian subject in need thereof. 2. The pharmaceutical formulation of claim 1, further comprising a lipid-based transfection reagent. 3. The pharmaceutical formulation of claim 1, wherein administration occurs at least once a day for 3 days. 4. The pharmaceutical formulation of claim 3, wherein administration occurs three times a day for 3 days. 5. The pharmaceutical formulation of claim 1, wherein the effective amount of mRNA is selected from the group consisting of 100 ng, 250 ng, 500 ng, 1000 ng and 1500 ng. 6. A pharmaceutical formulation comprising: 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; andiii) a pharmaceutically acceptable carrier,wherein the formulation is suitable for repeated administration to a mammalian subject in need thereof. 7. The pharmaceutical formulation of claim 6, further comprising a lipid-based transfection reagent. 8. The pharmaceutical formulation of claim 6, wherein administration occurs at least once a day for 3 days. 9. The pharmaceutical formulation of claim 8, wherein administration occurs three times a day for 3 days. 10. The pharmaceutical formulation of claim 6, wherein the effective amount of mRNA is selected from the group consisting of 100 ng, 250 ng, 500 ng, 1000 ng and 1500 ng. 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. 12. The mRNA sequence of claim 11, wherein the mRNA sequence comprises pseudouridine and 5-methyl-cytidine. 13. The mRNA sequence of claim 12, wherein the mRNA sequence comprising pseudouridine and 5-methyl-cytidine is SEQ ID NO: 20.
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