| abstract |
The present specification discloses a recognition element (iREMS) for intronic splicing modifiers that can be recognized by small molecule splicing modifier compounds of Formula (I) or forms thereof provided herein: [Formula I] Where W, X, A and B are as defined herein. In one aspect, the present disclosure discloses a method of altering RNA splicing to modulate the amount of product of a gene, wherein precursor RNA transcripts transcribed from a gene containing intronic REMS are spliced by formula (I). Modifiers are modified using compounds. In another aspect, the present disclosure discloses a method of altering RNA splicing to modulate the amount of an RNA transcript or protein product encoded by a gene, wherein the precursor RNA transcript transcribed from the gene is selected from the gene of Formula (I). It is altered to include intronic REMS using the flicting modifier compound. |