Compositions related to spherical nucleic acids (SNAs) with antisense oligonucleotides and methods of treatment of diseases and disorders are disclosed therein. In particular, the antisense oligonucleotides are targeted to a region in a pre-mRNA of interest to regulate pre-mRNA splicing.

The present invention provides an antisense oligonucleotide for inhibiting biosynthesis of chondroitin sulfate. The antisense oligonucleotide comprises at least one modified nucleotide, wherein the antisense oligonucleotide suppresses expression of one or both of the chondroitin sulfate N-acetylgalactosaminyltransferase-1 (CSGalNAcT1) gene and the chondroitin sulfate N-acetylgalactosaminyltransferase-2 (CSGalNAcT2) gene.

Described herein are aptamers capable of binding to human retinoic acid-inducible gene I protein (RIG-I); compositions comprising a RIG-I binding aptamer with a RIG-I; and methods of making and using the same.

Disclosed herein are antisense oligonucleotide sequences, and methods of use for treating neurological diseases. Described herein are oligonucleotide inhibitors. In various embodiments, the oligonucleotide targets a transcript for the treatment of neurological diseases, including motor neuron diseases, and/or neuropathies. For example, inhibitors of the transcript can be used to treat PD, ALS, FTD, and ALS with FTD.

The present disclosure relates to extracellular vesicles, e.g., exosomes, comprising an NLRP3 antagonist. In some aspects, the NLRP3 antagonist comprises an antisense oligonucleotide (ASO). Also provided herein are methods for producing the exosomes and methods for using the exosomes to treat and/or prevent diseases or disorders.

The invention provides an antisense oligonucleotide capable of controlling ATN1 gene expression and treating dentatorubral-pallidoluysian atrophy. An inventive compound comprises a modified oligonucleotide consisting of 8 to 80 linked nucleosides and having a nucleobase sequence including at least 8 contiguous nucleobases that are complementary to a transcript of ATN1, or a pharmacologically acceptable salt thereof.

Provided herein are compositions and methods for decreasing tau mRNA and protein expression. These compositions and methods are useful in treating tau-related diseases and disorders.

Disclosed herein are antisense compounds and methods for decreasing apo(a) to treat, prevent, or ameliorate diseases, disorders or conditions related to apo(a) or Lp(a). Certain diseases, disorders or conditions related to apo(a) or Lp(a) include inflammatory, cardiovascular and/or metabolic diseases, disorders or conditions. The antisense compounds disclosed herein can be used to treat such diseases, disorders or conditions in an individual in need thereof.

The present embodiments provide methods, compounds, and compositions useful for inhibiting PCSK9 expression, which may be useful for treating, preventing, or ameliorating a disease associated with PCSK9.

The present embodiments provide methods, compounds, and compositions for treating, preventing, or ameliorating a disease associated with excess growth hormone using antisense compounds or oligonucleotides targeted to growth hormone receptor (GHR).