A method of promoting the generation of oligodendrocytes from oligodendrocyte precursor cells by enhancing their survival and/or maturation includes administering to the cell an effective amount of an agent that enhances and/or induces accumulation of Δ8,9-unsaturated sterol intermediates of the cholesterol biosynthesis pathway in the oligodendrocyte precursor cells.

Aspects of the disclosure relate to complexes comprising a muscle-targeting agent covalently linked to a molecular payload. In some embodiments, the muscle-targeting agent specifically binds to an internalizing cell surface receptor on muscle cells. In some embodiments, the molecular payload promotes the expression or activity of a functional dystrophin protein. In some embodiments, the molecular payload is an oligonucleotide, such as an antisense oligonucleotide, e.g., an oligonucleotide that causes exon skipping in a mRNA expressed from a mutant DMD allele.

Disclosed herein are compositions and methods for the treatment of one or more progressive lung diseases, which may include, but are not limited to, chronic obstructive pulmonary disease (COPD), emphysema, and AAT deficient lung disease. The compositions useful for the disclosed methods may include an an antigen binding protein such as an anti-CELA1 antibody, an anti-CELA1 scFv, or an anti-CELA1 antisense nucleotide (ASO), which may be administered in an amount sufficient to treat one or more of the aforementioned disease states.

The present invention provides oligomeric compounds. Certain such oligomeric compounds are useful for hybridizing to a complementary nucleic acid, including but not limited, to nucleic acids in a cell. In certain embodiments, hybridization results in modulation of the amount activity or expression of the target nucleic acid in a cell.

The present invention discloses a method of treating, preventing or ameliorating tumor growth by administering a therapeutic agent that selectively inhibits dipeptidyl peptidase including fibroblast activation protein and dipeptidyl peptidase 8/9 in combination with an immune checkpoint inhibitor. The method specifically discloses use of Talabostat in combination with an immune checkpoint inhibitor, its pharmaceutical composition and process of preparing such composition.

Provided are compositions and methods for preventing, mitigating, decreasing, inhibiting and/or reversing bone loss and/or increasing and/or promoting bone regeneration and remodeling and/or preventing, mitigating, decreasing, inhibiting and/or reversing periodontitis and/or periodontal disease in a subject by administering to the subject an effective amount of an inhibitor of soluble epoxide hydrolase (sEH).

This disclosure relates to oligonucleotides, compositions and methods useful for reducing PCSK9 expression, particularly in hepatocytes. Disclosed oligonucleotides for the reduction of PCSK9 expression may be double-stranded or single-stranded, and may be modified for improved characteristics such as stronger resistance to nucleases and lower immunogenicity. Disclosed oligonucleotides for the reduction of PCSK9 expression may also include targeting ligands to target a particular cell or organ, such as the hepatocytes of the liver, and may be used to treat hypercholesterolemia, atherosclerosis, and/or one or more symptoms or complications thereof.

Provided herein are methods, compounds, and compositions for reducing expression of GYS1 in an individual. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate a glycogen storage disease or disorder in an individual in need.

The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the ALAS1 gene, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of ALAS1.

Described herein are compositions capable of targeting CaMKKβ and/or AMPK alpha and formulations thereof. Also described herein are methods of using the compositions and formulations thereof. In some embodiments, the compositions capable of targeting CaMKKβ and/or AMPK alpha and formulations thereof can prevent or treat outer hair cell loss, such as that which can occur as a result of auditory or chemical insult. In some embodiments, the compositions capable of targeting CaMKKβ and/or AMPK alpha and formulations thereof can treat or prevent acquired hearing loss.