US2008261277A1PendingUtilityA1
pRNA chimera
Est. expiryAug 23, 2020(expired)· nominal 20-yr term from priority
C12N 2320/32C12N 15/1131C12N 2310/53C12N 15/111C12N 15/1137C12N 2310/111C12N 2310/121C12N 2310/3519C12N 2310/11A61K 38/00C12N 2310/16A61P 31/00C12N 15/113A61P 35/00C12Y 113/11031C12N 2799/022
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Claims
Abstract
A circularly permuted chimeric pRNA molecule carrying a stabilized biologically active RNA, such as a ribozyme.
Claims
exact text as granted — not AI-modified1 . A pRNA chimera comprising a small interfering RNA (siRNA).
2 . The pRNA chimera of claim 1 comprising a base-paired region comprising the siRNA.
3 . The pRNA chimera of claim 1 which is circularly permuted.
4 . The pRNA chimera of claim 3 comprising a pRNA region and a spacer region, wherein the pRNA region comprises a based-paired region comprising a first biologically active nucleic acid comprising the siRNA, and the spacer region comprises a second biologically active nucleic acid.
5 . The pRNA chimera of claim 4 wherein the second biologically active nucleic acid comprises a therapeutic agent.
6 . The pRNA chimera of claim 4 wherein the second biological active nucleic acid possesses enzymatic activity or binding activity or both.
7 . The pRNA chimera of claim 4 wherein the second biologically active nucleic acid is selected from the group consisting of a ribozyme, an RNA aptamer, an antisense RNA and a peptide nucleic acid (PNA).
8 . The pRNA chimera of claim 1 which is not circularly permuted.
9 . The pRNA chimera of claim 1 comprising a 5′ or 3′ end comprising an end-labeling agent.
10 . The pRNA chimera of claim 9 wherein the end-labeling agent is selected from the group consisting of biotin, pCp, DIG, SH group and phosphate.
11 . The pRNA chimera of claim 1 comprising a 5′ or 3′ end comprising an exogenous RNA.
12 . The pRNA chimera of claim 1 comprising a 5′ or 3′ end comprising an RNA aptamer.
13 . The pRNA chimera of claim 1 comprising a 5′ or 3′ end comprising a therapeutic agent.
14 . The pRNA chimera of claim 1 comprising at least one nucleotide analog or modified nucleotide.
15 . The pRNA chimera of claim 1 wherein the nucleotide analog or modified nucleotide is selected from the group consisting of a DNA phosphorothoiate, an RNA phosphorothoiate and a 2′-O-methyl ribonucleotide.
16 . A DNA molecule comprising a nucleotide sequence that encodes a pRNA chimera comprising an siRNA.
17 . A method for making a pRNA chimera comprising:
providing a DNA encoding a pRNA chimera comprising an siRNA; and transcribing the DNA in vitro to yield the pRNA chimera.
18 . The method of claim 17 further comprising using polymerase chain reaction on a DNA template to generate the DNA encoding the pRNA chimera.
19 . The method of claim 17 further comprising generating the DNA encoding the pRNA chimera by cloning the DNA into a plasmid and replicating the plasmid.
20 . A method for delivering siRNA to a cell comprising:
introducing into the cell a DNA molecule comprising a nucleotide sequence that operably encodes the pRNA chimera comprising an siRNA; and causing transcription of the DNA to yield the siRNA.
21 . The method of claim 20 wherein the pRNA chimera is a circularly permuted pRNA chimera comprising a pRNA region and a spacer region, wherein the pRNA region comprises a based-paired region comprising a first biologically active nucleic acid comprising the siRNA, and the spacer region comprises a second biologically active nucleic acid.
22 . The method of claim 21 wherein the second biologically active RNA is selected from the group consisting of a ribozyme, an RNA aptamer, an antisense RNA and a peptide nucleic acid (PNA).
23 . The method of claim 20 wherein the cell is present in a cell culture, a tissue, an organ or an organism.
24 . The method of claim 20 wherein the cell is a mammalian cell.
25 . The method of claim 24 wherein the cell is a human cell.
26 . A polyvalent multimeric complex comprising a plurality of chimeric pRNA monomers comprising:
at least one chimeric pRNA monomer comprising a first biologically active nucleic acid comprising an siRNA; and at least one circularly permuted chimeric pRNA monomer comprising a pRNA region and a spacer region, wherein the spacer region comprises a second biologically active nucleic acid.
27 . The polyvalent multimeric complex of claim 26 wherein the second biologically active RNA is selected from the group consisting of a ribozyme, an RNA aptamer, an antisense RNA and a peptide nucleic acid (PNA).
28 . The polyvalent multimeric complex of claim 26 wherein the second biologically active RNA comprises an anti-receptor RNA.
29 . The polyvalent multimeric complex of claim 26 wherein at least one chimeric pRNA monomer comprises a 5′ or 3′ end comprising an end-labeling agent.
30 . The polyvalent multimeric complex of claim 29 wherein the end-labeling agent is selected from the group consisting of biotin, pCp, DIG, SH group and phosphate.
31 . The polyvalent multimeric complex of claim 26 wherein at least one chimeric pRNA monomer comprises a 5′ or 3′ end comprising an exogenous RNA.
32 . The polyvalent multimeric complex of claim 26 wherein at least one chimeric pRNA monomer comprises a 5′ or 3′ end comprising an RNA aptamer.
33 . The polyvalent multimeric complex of claim 26 wherein at least one chimeric pRNA monomer comprises a 5′ or 3′ end comprising a therapeutic agent.
34 . The polyvalent multimeric complex of claim 26 wherein at least one chimeric pRNA monomer comprises at least one nucleotide analog or modified nucleotide.
35 . The polyvalent multimeric complex of claim 34 wherein the nucleotide analog or modified nucleotide is selected from the group consisting of a DNA phosphorothoiate, an RNA phosphorothoiate and a 2′-O-methyl ribonucleotide.
36 . The polyvalent multimeric complex of claim 26 which is a pRNA hexamer.Join the waitlist — get patent alerts
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