Abstract: Described herein are oligonucleotides (e.g., single-stranded oligonucleotides) and compositions thereof for targeting a mutation in the spliceosome, such as the U 1 small nuclear RNA (snRNA), as well as related methods of use.

Abstract: The present disclosure features compounds of formula (1-a) and of formula (II) and pharmaceutical compositions thereof. The present disclosure further discloses the compounds of formula (I-a) and (II) and their compositions for use in methods of modulating nucleic acid splicing, e.g., splicing of a pre-mRNA, as well as said compounds for use in methods of treating e.g.: ?proliferative diseases, such as e.g. cancer, benign neoplasms or angiogenesis, ?neurological diseases or disorders, such as e.g. Huntington's disease, ?autoimmune diseases or disorders, immunodeficiency diseases or disorders, lysosomal storage disease or disorder, cardiovascular diseases or disorders, metabolic diseases or disorders, respiratory diseases or disorders, renal diseases or disorders, or infectious diseases. Exemplary compounds are e.g. 2-(indazol-5-yl)-6-(piperidin-4-yl)-1,7-naphthyridine derivatives and similar compounds.

Abstract: The present disclosure features compounds and related compositions that, inter alia, modulate nucleic acid splicing, e.g., splicing of a pre-mRNA, as well as methods of use thereof.

Abstract: The present disclosure features compounds of formula (1-a) and related compositions that, inter alia, modulate nucleic acid splicing e.g., splicing of a pre-mRNA, as well as methods of use thereof.

Abstract: Splice variants associated with neomorphic SF3B1 mutations are described herein. This application also relates to methods of detecting the described splice variants, and uses for diagnosing cancer, evaluating modulators of SF3B1, and methods of treating cancer associated with mutations in SF3B1.

Abstract: The present disclosure features compounds and related compositions that, inter alia, modulate nucleic acid splicing, e.g., splicing of a pre-mRNA, as well as methods of use thereof.

Abstract: The present disclosure features compounds and related compositions that, inter alia, modulate nucleic acid splicing, e.g., splicing of a pre-mRNA, as well as methods of use thereof.

Abstract: The present disclosure features compounds and related compositions that, inter alia, modulate nucleic acid splicing, e.g., splicing of a pre-mRNA, as well as methods of use thereof.

Abstract: The present disclosure features compounds and related compositions that, inter alia, modulate nucleic acid splicing, e.g., splicing of a pre-mRNA, as well as methods of use thereof.

Abstract: The present disclosure features compounds and related compositions that, inter alia, modulate nucleic acid splicing, e.g., splicing of a pre-mRNA, as well as methods of use thereof. Formula (I-b).

Abstract: Splice variants associated with neomorphic SF3B1 mutations are described herein. This application also relates to methods of detecting the described splice variants, and uses for diagnosing cancer, evaluating modulators of SF3B1, and methods of treating cancer associated with mutations in SF3B1.

Abstract: Splice variants associated with neomorphic SF3B1 mutations are described herein. This application also relates to methods of detecting the described splice variants, and uses for diagnosing cancer, evaluating modulators of SF3B1, and methods of treating cancer associated with mutations in SF3B1.

Abstract: Splice variants associated with neomorphic SF3B1 mutations are described herein. This application also relates to methods of detecting the described splice variants, and uses for diagnosing cancer, evaluating modulators of SF3B1, and methods of treating cancer associated with mutations in SF3B1.