The disclosure provides a composition for suppressing inflammation comprising at least one substance that disrupts a stem-loop structure in the 3′ untranslated region of a Regnase-1 mRNA, wherein the stem-loop structure is at least one stem-loop structure selected from a first stem-loop structure formed in a region corresponding to positions 231 to 245 of SEQ ID NO: 1 and a second stem-loop structure formed in a region corresponding to positions 424 to 442 of SEQ ID NO: 1.

The present description relates to the use of a SRSF3 agent for regulating the function of a myeloid cell, such as a microglial cell and/or monocyte, for treating neurological conditions, cancers, bacterial infections and viral infections wherein the SRSF3 agent inhibits expression or function of SRSF3.

The present invention relates to the treatment of muscular diseases.

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.

The present disclosure relates to double-stranded oligonucleotides, including double-stranded oligonucleotides such as siRNAs, comprising a sense strand oligonucleotide and an antisense strand oligonucleotide, and wherein the antisense strand oligonucleotide comprises one or more nucleotide analogs of formula (I-A) which are neither the 5′-overhang nucleotide nor the 3′-overhang nucleotide of the said antisense strand oligonucleotide, and wherein a nucleotide analog of formula (I-A) is as described in the disclosure. Oligonucleotides containing these analogs have superior biological activity, for example, increased in vitro stability and improved in vivo potency especially improved off-target profiles. The improved oligonucleotides are useful for silencing (e.g., reducing or eradicating) the expression of a target gene.

Provided herein are antisense oligonucleotides (ASOs) specific for Rab13 and Net1, for example specific for a GA-rich region of the 3′-UTR. In some examples, the ASOs are modified. Methods of using these ASOs to reduce the migration of metastatic cancer cells are provided, for example as a use to treat metastatic cancer.

Antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon 53 skipping are described.

The present disclosure relates to modified antisense oligonucleotides. The nucleotides described herein are of 10 to 40 nucleobases and include a targeting sequence complementary to a target region within intron 1 of a pre-mRNA of the human alpha glucosidase (GAA) gene. The target region includes at least one additional nucleobase compared to the targeting sequence, wherein the at least one additional nucleobase has no complementary nucleobase in the targeting sequence, and wherein the at least one additional nucleobase is internal to the target region.

Antisense oligomers complementary to a selected target site in the human dystrophin gene to induce exon 2 skipping are described. In various aspects, antisense oligomers are described according to Formula (I): Formula (I) or a pharmaceutically acceptable salt thereof, wherein T, Nu, n, and R.sup.100 are defined herein.

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The present invention concerns the use of antisense oligonucleotides (AON) capable of inhibiting expression of dynamin 2, advantageously human dynamin 2, for use in the treatment of Charcot-Marie-Tooth disease (CMT) and centronuclear myopathies (CNM).