The present invention relates the treatment and prognostic of cancer like glioblastoma. Here, the inventors focused their study on the impact of presence of N6-adenosine methylation in miRNA-200b-3p in samples of patients suffering from glioblastoma multiforme (GBM). Their study was particularly focused on the impact of miRNA-200b-3p and its adenosine methylation on the expression of XIAP. XIAP acts as an anti-apoptotic protein via the inhibition of caspase-3 and -7 activation and high XIAP expression is associated with a poor survival in several solid tumors. Thus, the miR-200b-3p-mediated repression of XIAP mRNA expression appears as a mechanism governing the caspase-3 and -7 activity and the apoptosis. In theory, in the presence of miR-200b-3p, XIAP mRNA expression is repressed and caspase-3 and -7 can be activated to promote apoptosis. Thus, the present invention relates to an in vitro method for determining the prognosis of the survival time of a patient suffering from a cancer comprising the steps consisting of i) determining the expression level of the miR-200b-3p and/or the N6-adenosine methylated miRNA-200b-3p (miR-200b-3p m6A) in a sample from said patient and to the N6-adenosine methylated miRNA-200b-3p (miR-200b-3p m6A) for use in the treatment of a cancer in a subject in need thereof.

The invention provides immunomodulatory polynucleotides and methods for immunomodulation of individuals using the immunomodulatory polynucleotides.

Described are RNAi agents, compositions that include RNAi agents, and methods for inhibition of an alpha-ENaC (SCNN1A) gene. The alpha-ENaC RNAi agents and RNAi agent conjugates disclosed herein inhibit the expression of an alpha-ENaC gene. Pharmaceutical compositions that include one or more alpha-ENaC RNAi agents, optionally with one or more additional therapeutics, are also described. Delivery of the described alpha-ENaC RNAi agents to epithelial cells, such as pulmonary epithelial cells, in vivo, provides for inhibition of alpha-ENaC gene expression and a reduction in ENaC activity, which can provide a therapeutic benefit to subjects, including human subjects.

Described herein are small molecule splicing modulator compounds that modulate splicing of mRNA, such as pre-mRNA, encoded by genes, and methods of use of the small molecule splicing modulator compounds for modulating splicing and treating diseases and conditions.

The present invention provides an aptamer that binds to chymase, and contains a common sequence represented by UAACR.sub.1N.sub.1R.sub.2GGGG wherein R.sub.1 and R.sub.2 are each any one base, and N.sub.1 shows 3 to 30 bases (uracil is optionally thymine).

The current invention provides an improved oligonucleotide and its use for treating, ameliorating, preventing, delaying and/or treating a human cis-element repeat instability associated genetic neuromuscular or neurodegenerative disorder.

Described herein are small molecule splicing modulator compounds that modulate splicing of mRNA, such as pre-mRNA, encoded by genes, and methods of use of the small molecule splicing modulator compounds for modulating splicing and treating diseases and conditions.

Disclosed herein are compositions that comprise engineered polynucleotides, pharmaceutical compositions comprising the same, methods of making the same, and methods of treatment comprising the compositions that comprise the engineered polynucleotides.

The invention relates to functional nucleic acid molecules comprising a target determinant sequence and a regulatory sequence wherein the functional nucleic acid molecule comprises one or more chemical modifications, particularly for use in methods of increasing target protein synthesis efficiency.

Described herein are small molecule splicing modulator compounds that modulate splicing of mRNA, such as pre-mRNA, encoded by genes, and methods of use of the small molecule splicing modulator compounds for modulating splicing and treating diseases and conditions.