Claims for Patent: 9,434,947
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Summary for Patent: 9,434,947
Title: | Modulation of KCNH2 isoform expression by oligonucleotides as a therapeutic approach for long QT syndrome |
Abstract: | Oligonucleotides with activity in preventing poly(A) adenylation at intron 9 of the KCNH2 gene, as well as pharmaceutical compositions comprising the oligonucleotides and methods of using the oligonucleotides to treat long QT syndrome in a subject are disclosed. The oligonucleotides include antisense sequences corresponding to sites termed DSE-1 and DSE-2 in intron 9. |
Inventor(s): | Zhou; Zhengfeng (Portland, OR), Gong; Qiuming (Portland, OR), Stump; Matthew (Portland, OR) |
Assignee: | OREGON HEALTH & SCIENCE UNIVERSITY (Portland, OR) |
Application Number: | 14/600,958 |
Patent Claims: | 1. An isolated oligonucleotide comprising SEQ ID NO: 4 that inhibits the formation of a poly(A) signal in intron 9 of KCNH2, wherein the oligonucleotide is no more
than 30 nucleotides in length and wherein the oligonucleotide comprises a modified nucleotide, a locked nucleotide, a G-clamp nucleotide, a nucleotide base analog, a 3'-terminal cap moiety, or a phosphate backbone modification.
2. The oligonucleotide of claim 1 comprising SEQ ID NO: 5. 3. The oligonucleotide of claim 2 consisting of a sequence of SEQ ID NO: 5. 4. The oligonucleotide of claim 1 comprising a morpholino nucleotide. 5. The oligonucleotide of claim 4 wherein all of the nucleic acids in the oligonucleotide are morpholino nucleotides. 6. A pharmaceutical composition comprising an effective amount of the oligonucleotide of claim 1 and a pharmaceutically acceptable carrier. 7. A method of treating long-QT syndrome caused by mutations in the KCNH2 gene, the method comprising: administering to the subject the pharmaceutical composition of claim 6, thereby treating the long-QT syndrome. |
Details for Patent 9,434,947
Applicant | Tradename | Biologic Ingredient | Dosage Form | BLA | Approval Date | Patent No. | Expiredate |
---|---|---|---|---|---|---|---|
Merck Sharp & Dohme Corp. | INTRON A | interferon alfa-2b | For Injection | 103132 | 06/04/1986 | ⤷ Try a Trial | 2039-02-26 |
Merck Sharp & Dohme Corp. | INTRON A | interferon alfa-2b | For Injection | 103132 | ⤷ Try a Trial | 2039-02-26 | |
Merck Sharp & Dohme Corp. | INTRON A | interferon alfa-2b | Injection | 103132 | ⤷ Try a Trial | 2039-02-26 | |
>Applicant | >Tradename | >Biologic Ingredient | >Dosage Form | >BLA | >Approval Date | >Patent No. | >Expiredate |
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