摘要:
Compounds, compositions and methods are provided for modulating the RNAi pathway. The compositions comprise oligonucleotides, targeted to nucleic acid molecules encoding EIF2C2. Methods of using these compounds for modulation of RNA interference as well as EIF2C2 expression and for diagnosis and treatment of disease associated with expression of EIF2C2 are provided. Also provided are compounds, compositions and methods for modulating the expression of DDX36. The compositions comprise oligonucleotides, targeted to nucleic acid encoding DDX36. Methods of using these compounds for modulation of DDX36 expression and for diagnosis and treatment of diseases and conditions associated with expression of DDX36 are provided.
摘要:
Compositions and methods are provided for the modulation of expression of cellular adhesion molecules. In accordance with preferred embodiments, oligonucleotides are provided which are specifically hybridizable with nucleic acids encoding intercellular adhesion molecule-1, vascular cell adhesion molecule-1, and endothelial leukocyte adhesion molecule-1. Methods of modulating expression of cellular adhesion molecules are provided, as are methods of treating conditions associated with cellular adhesion molecules. In a preferred embodiment, the cellular adhesion molecule is ICAM-1, and a preferred antisense sequence targeted to human ICAM-1 is demonstrated to have clinical utility in several disease indications.
摘要:
Compounds, compositions and methods are provided for modulating the expression of SLC26A2. The compositions comprise oligonucleotides, targeted to nucleic acid encoding SLC26A2. Methods of using these compounds for modulation of SLC26A2 expression and for diagnosis and treatment of disease associated with expression of SLC26A2 are provided.
摘要:
The present invention provides double stranded compositions that have a region that is complementary to a target nucleic acid. The targeting strand comprises linked ribofuranosyl nucleosides and the second strand comprises linked modified nucleosides that have 3′-endo conformational geometry. The strands can be linked together or separate and may contain additional groups. The present invention also provides methods of using the compositions for modulating gene expression.
摘要:
Antisense compounds, compositions and methods are provided for modulating the expression of TFAP2C. The compositions comprise antisense compounds, particularly antisense oligonucleotides, targeted to nucleic acids encoding TFAP2C. Methods of using these compounds for modulation of TFAP2C expression and for treatment of diseases associated with expression of TFAP2C are provided.
摘要:
The present invention provides methods of identifying an oligomeric compound having bioactivity in vivo, methods of identifying a small interfering RNA having bioactivity in vivo, and kits.
摘要:
Compositions and methods are provided for inhibiting the expression of human tumor necrosis factor-α (TNF-α). Antisense oligonucleotides targeted to nucleic acids encoding TNF-α are preferred. Methods of using these oligonucleotides for inhibition of TNF-α expression and for treatment of diseases, particularly inflammatory and autoimmune diseases, associated with overexpression of TNF-α are provided.
摘要:
Compounds, compositions and methods are provided for modulating the expression of splice variants or products. The compositions comprise oligonucleotides, targeted to nucleic acid encoding splice variants or products. Methods of using these compounds for modulation of expression of splice variants or products and for diagnosis and treatment of disease associated with expression of splice variants or products are provided.
摘要:
Interative, preferably computer based iterative processes for generating synthetic compounds with desired physical, chemical and/or bioactive properties, i.e., active compounds, are provided. During iterations of the processes, a target nucleic acid sequence is provided or selected, and a library of candidate nucleobase sequences is generated in silico according to defined criteria. A “virtual” oligonucleotide chemistry is chosen and a library of virtual oligonucleotide compounds having the selected nucleobase sequences is generated. These virtual compounds are reviewed and compounds predicted to have particular properties are selected. The selected compounds are robotically synthesized and are preferably robotically assayed for a desired physical, chemical or biological activity. Active compounds are thus generated and, at the same time, preferred sequences and regions of the target nucleic acid that are amenable to oligonucleotide or sequence-based modulation are identified.
摘要:
Compositions and methods are provided for inhibiting the translation of a capped target mRNA. Antisense oligomers of the invention are targeted to the 5' cap region of the target mRNA and include oligonucleosides, PNAs, or oligonucleotides modified at the 2' position of the sugar. Preferably said oligomers inhibit protein translation directly via interference with ribosome assembly.