The present disclosure features useful compositions and methods to recruit RNA editing enzymes and treat disorders for which deamination of an adenosine in an mRNA produces a therapeutic result, e.g., in a subject in need thereof.

The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to synthetic chemically modified small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.

The invention provides a double-stranded ribonucleic acid (dsRNA) having a chain length suitable for simultaneously showing low toxicity and high function in the use of an adjuvant and the like, and resisting variation of chain length even when subjected to a heating and cooling treatment, or a salt thereof; an immune potentiator, adjuvant, pharmaceutical product and the like containing the dsRNA and the like; and a production method of such dsRNA and the like. The invention is characterized in that the weight average chain length of two or more single-stranded ribonucleic acids (ssRNAs) constituting the first chain constituting dsRNA is not more than ½ of the weight average chain length of one ssRNA constituting the second chain.

This invention relates to compounds, compositions, and methods useful for reducing KRAS target RNA and protein levels via use of Dicer substrate siRNA (DsiRNA) agents possessing asymmetric end structures.

The invention provides compositions and methods for reducing expression of a target gene in a cell, involving contacting a cell with an isolated double stranded nucleic acid (dsNA) in an amount effective to reduce expression of a target gene in a cell. The dsNAs of the invention possess a single stranded extension (in most embodiments, the single stranded extension comprises at least one modified nucleotide and/or phosphate back bone modification). Such single stranded extended Dicer-substrate siRNAs (DsiRNAs) were demonstrated to be effective RNA inhibitory agents compared to corresponding double stranded DsiRNAs.

The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of modulatory polynucleotides.

The invention provides compositions and methods for reducing expression of a target gene in a cell, involving contacting a cell with an isolated double stranded nucleic acid (dsNA) in an amount effective to reduce expression of a target gene in a cell. The dsNAs of the invention possess a single stranded extension (in most embodiments, the single stranded extension comprises at least one modified nucleotide and/or phosphate back bone modification). Such single stranded extended Dicer-substrate siRNAs (DsiRNAs) were demonstrated to be effective RNA inhibitory agents compared to corresponding double stranded DsiRNAs.

This invention relates to compounds, compositions, and methods useful for reducing lactate dehydrogenase target RNA and protein levels via use of ds RNAs, e.g., Dicer substrate siRNA (DsiRNA) agents.

Provided herein are double-stranded nucleic acid inhibitor molecules having a shortened sense strand with a stem loop structure and an antisense strand. Also provided are methods and compositions for reducing target gene expression and methods and compositions for treating a disease of interest.

This disclosure relates to the use of RNA oligonucleotides, compositions and methods useful for reducing ALDH2 or other target gene expression, in the central nervous system. In some embodiments, the oligonucleotide is used in methods of treating neurological diseases. Stable oligonucleotide derivatives that have enhanced activity in the central nervous system are provided.