摘要:
Short interfering ribonucleic acid (siRNA) for oral administration, said siRNA comprising two separate RNA strands that are complementary to each other over at least 15 nucleotides, wherein each strand is 49 nucleotides or less, and wherein at least one of which strands contains at least one chemical modification.
摘要:
The invention provides immunostimulatory compositions and methods for their use. In particular, the immunostimulatory compositions of the invention include RNA-like polymers that incorporate an immunostimulatory sequence motif and at least one chemical modification to confer improved stability against nuclease degradation and improved activity. Specific modifications involving phosphate linkages, nucleotide analogs, and combinations thereof are provided. Compositions of the invention optionally include an antigen and can be used to stimulate an immune response. Also provided are compositions and methods useful for treating a subject having an infection, a cancer, an to allergic condition, or asthma. Modified oligoribonucleotide analogs of the invention are believed to stimulate Toll-like receptors TLR7 and TLR8.
摘要:
The present invention discloses antisense poly-2′-O-(2,4-dinitrophenyl) oligoribonucleotides which are capable of down regulating the expression of the RIα subunit of protein kinase A. An example is 5′-GGCUGCGUGCCUCCUCACUGG (named antisense poly-DNP RNA-21) or a sequence which has a one-base mismatch therewith. The antisense oligoribonucleotide can be synthesized by in vitro transcription followed by chemical derivatization. The base sequence of the oligoribonucleotides is complementary to that of nt 110 to 130 in RIα/PKA mRNA. The antisense poly-DNP RNA-21 was found to inhibit cell growth with IC50 values in the nanomolar range. These oligonucleotides can be used as effective anti-cancer agents.
摘要:
The present invention provides antisense oligoribonucleotides with sequences that recognize and bind to the c-myc gene or c-myc mRNA, where one or more sugar residues of the oligoribonucleotides are modified by the substitution of DNP at the 2′-O position. The antisense oligoribonucleotides can be used for inhibiting the growth of cells in which the c-myc gene is overexpressed by down-regulation of transcription of c-myc RNA or translation of the c-myc protein. The antisense oligoribonucleotides can also be used for diagnostic purposes to identify the overexpression of the c-myc gene. The nucleotide sequence of the DNP-oligoribonucleotides is complementary to the sequence 578 to 598 in c-myc mRNA and does not contain a G-quartet motif. This DNP-RNA can silence c-myc gene expression both in vitro and in vivo with high efficacy and sequence specificity.
摘要:
A method of modulating the expression of a nucleic acid in the hepatic system of a mammal is disclosed. In addition, a method of preferentially targeting an oligonucleotide to liver cells in a mammal is disclosed. Also, a method of treating an animal having a hepatic disease or disorder associated with a protein encoded by a gene is disclosed. A composition containing an oligonucleotide is disclosed.