The present invention relates to compositions of recombinant polynucleic acid constructs comprising at least one nucleic acid sequence encoding an siRNA capable of binding to a target mRNA and at least one nucleic acid sequence encoding a gene of interest. Also disclosed herein is use of the compositions in treating a disease or a condition and in simultaneously modulating expression of two or more genes.

siRNA compositions are provided that contain gemcitabine (GEM) in place of cytosine moieties within the siRNA sequence. Pharmaceuticals compositions containing these siRNA molecules, and methods of using the compositions for treating diseases such as cancer are provided.

Disclosed is a method for treating endometriosis, preventing endometriosis, or ameliorating a symptom of endometriosis in a subject comprising the step of administering to the subject a therapeutically effective amount of a pharmaceutical composition comprising as an active ingredient a therapeutic dsRNA (tdsRNA).

Disclosed is a method for treating a cancer, such as pancreatic cancer, in a subject. The method comprises a first step of administering to the subject a standard of care therapy such as FOLFIRINOX and administering to the subject a therapeutic double stranded RNA (tdsRNA) which can be Rintatolimod.

The present invention relates to a method for providing information on the diagnosis of Parkinson's disease. The present invention also relates to a composition for preventing, ameliorating or treating Parkinson's disease. The present invention uses at least one miRNA whose expression is specifically down- or up-regulated in a Parkinson's disease model. Therefore, the use of the miRNA is effective in diagnosing and treating Parkinson's disease.

The present disclosure relates to methods of treating heat shock factor 1 (HSF1)-related diseases such as cancer and viral diseases, using a therapeutically effective amount of a RNAi agent to HSF.

The present invention provides an oligonucleotide comprising the sequence 5′-GGAACAGTTCGTCCATGGC-3′ (SEQ ID NO:2) for use in the treatment of an inflammatory bowel disease in a human subject via topical administration to the colon, wherein the subject has not been subjected to colonic cleaning prior to said administration.

Provided herein are DNA aptamers targeting AXL receptor kinase. The DNA aptamers may comprise a thiophosphate backbone and be chemically modified. Further provided herein are methods of use thereof for the treatment of a disease or disorder, such as cancer.

Compositions and methods for treating diseases and conditions mediated by the high affinity IgE receptor (FcεRI) are provided. Also provided are antisense oligomers for modulating splicing of mRNA encoding a MS4A6A protein, optionally in addition to antisense oligomers for modulating splicing of mRNA encoding the FcεRIβ protein, thereby down-regulating cell-surface expression of FcεRI, and uses of the antisense oligomers for inhibiting mast cell degranulation, cytokine release, migration, and proliferation; for inhibiting anaphylaxis reactions in individuals, for treating allergic conditions in individuals, for reducing the incidence of allergic reactions in individuals, for treating individuals at risk of developing anaphylactic reactions, and for treating mast cell-related diseases in individuals.

RNAs, such as miRNA and siRNA, and their use in treating complement-mediated disorders, are described.