摘要:
Methods for slowing disease progression in an individual suffering from familial ALS are provided. Also provided are methods of increasing the survival time of an individual suffering from familial ALS. These methods employ antisense oligonucleotides targeted to SOD1, for use in inhibiting the expression of SOD1 in the central nervous system of an individual suffering from familial ALS.
摘要:
Modified oligonucleotides having a conserved G4 sequence and a sufficient number of flanking nucleotides to significantly inhibit the activity of a virus or phospholipase A2 or to modulate the telomere length of a chromosome are provided. G4 quartet oligonucleotide structures are also provided. Methods of prophylaxis, diagnostics and therapeutics for viral-associated diseases and diseases associated with elevated levels of phospholipase A2 are also provided. Methods of modulating telomere length of a chromosome are also provided; modulation of telomere length is believed to play a role in the aging process of a cell and in control of malignant cell growth.
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:
Antisense compounds, compositions and methods are provided for modulating the expression of Integrin beta 4 binding protein. The compositions comprise antisense compounds, particularly antisense oligonucleotides, targeted to nucleic acids encoding Integrin beta 4 binding protein. Methods of using these compounds for modulation of Integrin beta 4 binding protein expression and for treatment of diseases associated with expression of Integrin beta 4 binding protein are provided.
摘要:
Antisense compounds, compositions and methods are provided for modulating the expression of CD40 ligand. The compositions comprise antisense compounds, particularly antisense oligonucleotides, targeted to nucleic acids encoding CD40 ligand. Methods of using these compounds for modulation of CD40 ligand expression and for treatment of diseases associated with expression of CD40 ligand are provided.
摘要:
The present invention provides methods of identifying oligomeric compounds, such as siRNA and double-stranded RNA compounds, having bioactivity in vivo, and kits.
摘要:
The present invention provides methods for the identification of target molecules that bind to ligands, particularly microRNA ligands and mimics thereof and/or microRNA target molecules and mimics thereof, with as little as millimolar (mM) affinity using mass spectrometry. The methods may be used to determine the mode of binding interaction between two or more of these target molecules to the ligand as well as their relative affinities. Also provided are methods for designing compounds having greater affinity to a ligand by identifying two or more target molecules using mass spectrometry methods of the invention and linking the target molecules together to form a novel compound.
摘要:
The present invention provides compositions and methods for reducing levels of a preselected mRNA, using antisense compounds targeted to a splice site or a region up to 50 nucleobases upstream of an exon/intron junction on said mRNA. Preferably, said antisense compounds do not elicit RNAse H cleavage of the mRNA.
摘要:
Anti sense oligonucleotide compositions which specifically hybridize with nucleic acids encoding B7 proteins, and use of these compositions for inhibiting expression of B7 mRNA.