The present invention provides antisense oligonucleotides directed against immune checkpoints and methods and compositions of using such antisense oligonucleotides for the treatment of cancer.

A chimeric complex comprising a microRNA in combination with an aptamer for AXL receptor tyrosine kinase is provided. Use of the chimeric complex for targeted treatment of a tumor disease, in particular in a therapy affecting onset and/or progression of tumor metastasis is also provided.

Disclosed are methods and compositions for at least preventing, treating, inhibiting, or attenuating an Ebola virus infection of a subject. The methods comprise administering an effective amount of a composition as described herein to the subject thereby at least preventing, treating, inhibiting, or attenuating the Ebola virus infection of the subject. The compositions comprise a therapeutic double-stranded RNA (tdsRNA) and additional optional components such as an Ebola antigen.

The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting a SOD1 gene, as well as methods of inhibiting expression of a SOD1 gene and methods of treating subjects having a SOD1-associated neurodegenerative disease or disorder, e.g., Amyotrophic Lateral Sclerosis (ALS), Alzheimer's disease (AD), Parkinson's disease (PD), and Down's syndrome (DS), using such dsRNAi agents and compositions.

miRNAs for in vitro diagnosis of resistance of tumors to BRAF/MEK pathway (also named as MAPK 5 pathway) inhibiting drugs and for treatment of tumors which are treated with said drugs, such as melanoma, by stimulating or inhibiting the expression of down-regulated or up-regulated miRNAs, respectively.

The invention provides oligonucleotides complementary to a non-coding chimeric mitochondrial RNA as well as compositions and kits comprising the same, and their use in treating and preventing metastasis or relapse of a cancer in an individual previously treated for cancer with a therapy. The invention also provides oligonucleotides complementary to a non-coding chimeric mitochondrial RNA as well as compositions and kits comprising the same, and their use in treating a refractory cancer (e.g., a refractory HPV-associated cancer).

Provided herein are methods and compositions for treating cancer, including cancer that is not responsive to immunotherapy. In one aspect, the methods of treatment comprise administering to the subject a therapeutically effective amount of a β-catenin inhibitor and a therapeutically effective amount of an immunotherapeutic agent. Another aspect is directed to pharmaceutical compositions comprising a β-catenin inhibitor for use in treating cancer, wherein the composition is administered in combination with an immunotherapeutic agent. Yet another aspect is directed to a method of potentiating the therapeutic effect of immunotherapy against a cancer using a β-catenin inhibitor, such as a β-catenin nucleic acid inhibitor molecule.

Inhibitory RNA molecules that specifically inhibit mammalian RIZ2 expression, with therapeutic effect in cell proliferative diseases, such as cancer.

A method of inhibiting growth of a bacterium including steps of: a) delivering a polynucleotide including a sequence of SEQ ID NO: 1 or a homologue thereof to the bacterium, and b) contacting the bacterium with an effective amount of an antibiotic; wherein the bacterium is Pseudomonas aeruginosa. A method of treating a subject suffering from an infection caused by a bacterium which is Pseudomonas aeruginosa and the method including steps of: i) delivering said polynucleotide to a tissue infected by the bacterium or a bacterial cell in the subject; and ii) administering an effective amount of an antibiotic to the subject. A composition and a recombinant plasmid containing said polynucleotide.

Compositions and methods for editing, e.g., introducing double-stranded breaks, within the TTR gene in combination with administration of a corticosteroid are provided. Compositions and methods for treating subjects having amyloidosis associated with transthyretin (ATTR), in which a guide RNA and a corticosteroid are administered, are provided.